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  • 2024/bielefeld-cebitec
  • l-sanfilippo/bielefeld-ce-bi-tec-temp
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src/pages.ts
View file @ 3fd292fd
......@@ -2,11 +2,9 @@
Contribution,
Description,
Engineering,
Experiments,
Home,
Attributions,
HumanPractices,
Supplementary,
Parts,
Judging,
ProDesc,
......@@ -22,7 +20,7 @@
SafetySidebar
} from "./contents";
import { DescSidebar, NoSidebar, EngSide } from "./contents";
import { IBIE, JUDGEH, PRODESC, SUPH, PARTH, ENGH, HOMEH, HPH, SPONH, RESH, ATTH, CONTH, DESCH, EXPH, IMPH, NOTEH, SAFEH, TEAMH } from "./contents";
import { IBIE, JUDGEH, PRODESC, PARTH, ENGH, HOMEH, HPH, SPONH, RESH, ATTH, CONTH, DESCH, IMPH, NOTEH, SAFEH, TEAMH } from "./contents";
import { Methods } from "./contents/methods";
import { METHH } from "./headers/meth-h";
import { ConSidebar } from "./sidebars/conS";
......@@ -100,14 +98,6 @@ const Pages: (Page | Folder)[] = [
header: HOMEH,
navlist: NoSidebar,
},
{
name: "Experiments",
title: "Experiments",
path: "/experiments",
component: Experiments,
header: EXPH,
navlist: NoSidebar,
},
{
name: "Human Practices",
title: "Human Practices",
......@@ -221,14 +211,6 @@ const Pages: (Page | Folder)[] = [
header: SAFEH,
navlist: SafetySidebar,
},
{
name: "Supplementary Material",
title: "Supplementary",
path: "/supplement",
component: Supplementary,
header: SUPH,
navlist: NoSidebar,
},
{
name: "Roster",
title: "Roster",
......@@ -244,10 +226,7 @@ export const NavPages: (Page | PageRef | Folder)[] = [
{
name: "Home",
title: "Bielefeld CeBiTec",
path: "/home?=scrollTo=l&ing-page-header",
component: Home,
header: HOMEH,
navlist: NoSidebar,
path: "/home?=scrollTo=lpbild",
},
{
name: "Highlights",
......@@ -260,22 +239,22 @@ export const NavPages: (Page | PageRef | Folder)[] = [
{
name: "Engineering",
title: "Engineering",
path: "/engineering"
path: "/engineering?tab=tab-our-cycle&scrollTo=ourcycle"
},
{
name: "Experiments",
title: "Experiments",
path: "/experiments?tab=heading"
name: "Materials & Methods",
title: "Materials & Methods",
path: "/materials-methods?scrollTo=introduction"
},
{
name: "Results",
title: "Results",
path: "/results?scrollTo=experimental-design"
path: "/results?scrollTo=abstract"
},
{
name: "Parts",
title: "Parts",
path: "/parts?scrollTo=Parts Collection2H"
path: "/parts?scrollTo=parts-collection"
},
{
name: "Judging",
......@@ -291,33 +270,21 @@ export const NavPages: (Page | PageRef | Folder)[] = [
name: "Roster",
title: "Roster",
path: "/team",
component: Team,
header: TEAMH,
navlist: NoSidebar,
},
{
name: "Attributions",
title: "Attributions",
path: "/attributions",
component: Attributions,
header: ATTH,
navlist: NoSidebar,
},
{
name: "Sponsors & Partners",
title: "Sponsors & Partners",
path: "/partners",
component: Partners,
header: SPONH,
navlist: NoSidebar,
path: "/partners?scrollTo=gold",
},
{
name: "iGEM Bielefeld",
title: "iGEM Bielefeld",
path: "/igem-bielefeld",
component: igemBielefeld,
header: IBIE,
navlist: iGemBielefeldSidebar,
path: "/igem-bielefeld?scrollTo=bielefeld-university",
},
],
},
......@@ -327,84 +294,47 @@ export const NavPages: (Page | PageRef | Folder)[] = [
{
name: "Description",
title: "Project Description",
path: "/description",
component: Description,
header: DESCH,
navlist: DescSidebar
path: "/description?scrollTo=Abstract",
},
{
name: "Engineering",
title: "Engineering",
path: "/engineering",
component: Engineering,
header: ENGH,
navlist: EngSide ,
path: "/engineering?tab=tab-our-cycle&scrollTo=ourcycle",
},
{
name: "Biosafety",
title: "Biosafety",
path: "/safety",
component: Safety,
header: SAFEH,
navlist: SafetySidebar,
path: "/safety?scrollTo=role-in-igem",
},
{
name: "Project Documentation",
title: "Project Documentation",
path: "/project-documentation",
component: ProDesc,
header: PRODESC,
navlist: NoSidebar
},
{
name: "Supplementary Material",
title: "Supplementary",
path: "/supplement",
component: Supplementary,
header: SUPH,
navlist: NoSidebar,
},
path: "/project-documentation?scrollTo=our-meeting-protocols",
}
],
},
{
name: "Lab",
folder: [
{
name: "Experiments",
title: "Experiments",
path: "/experiments",
component: Experiments,
header: EXPH,
navlist: NoSidebar,
},
{
name: "Materials & Methods",
title: "Materials & Methods",
path: "/materials-methods",
component: Methods,
header: METHH,
navlist: MethSidebar,
path: "/materials-methods?scrollTo=introduction",
},
{
name: "Results",
title: "Results",
path: "/results",
component: Results,
header: RESH,
navlist: ResultSidebar,
path: "/results?scrollTo=abstract",
},
{
name: "Parts",
title: "Parts",
path: "/parts",
path: "/parts?scrollTo=Description1H",
},
{
name: "Notebook",
title: "Notebook",
path: "/notebook",
component: Notebook,
header: NOTEH,
navlist: NoSidebar,
path: "/notebook?scrollTo=notebookH",
},
]
},
......@@ -419,10 +349,7 @@ export const NavPages: (Page | PageRef | Folder)[] = [
{
name: "Integrated Human Practices",
title: "Integrated Human Practices",
path: "/human-practices",
component: HumanPractices,
header: HPH,
navlist: HpSidebar
path: "/human-practices?scrollTo=overview-stakeholders",
},
{
name: "Feedback & Implementation",
......@@ -460,9 +387,9 @@ export const NavPages: (Page | PageRef | Folder)[] = [
path: "/judging?scrollTo=Overview",
},
{
name: "Best New Basic Part",
name: "Best Integrated Human Practice",
title: "Judging",
path: "/judging?scrollTo=best-new-basic-part",
path: "/judging?scrollTo=best-integrated-human-practice",
},
{
name: "Safety & Security",
......@@ -470,10 +397,15 @@ export const NavPages: (Page | PageRef | Folder)[] = [
path: "/judging?scrollTo=safety-security",
},
{
name: "Best Integrated Human Practice",
name: "Best New Basic Part",
title: "Judging",
path: "/judging?scrollTo=best-integrated-human-practice",
},
path: "/judging?scrollTo=best-new-basic-part",
},
{
name: "Judging Session",
title: "Judging",
path: "/judging?scrollTo=judging-session",
},
]
},
{
......@@ -482,35 +414,22 @@ export const NavPages: (Page | PageRef | Folder)[] = [
{
name: "Contribution",
title: "Contribution",
path: "/contribution",
component: Contribution,
header: CONTH,
navlist: ConSidebar,
path: "/contribution?scrollTo=abstract",
},
{
name: "BFH European MeetUp",
title: "MeetUp",
path: "contribution/?scrollTo=BFH European MeetUp"
path: "contribution/?scrollTo=bfh-european-meetup"
},
{
name: "Safety Guideline",
title: "Safety Guideline",
path: "contribution/?scrollTo=primary-culture-safety-guideline"
name: "Our Contributions",
title: "Our Contributions",
path: "contribution/?scrollTo=our-contributions"
},
{
name: "Collaborations",
title: "Collaborations",
path: "contribution/?scrollTo=collaborations"
},
{
name: "Patient Matters",
title: "Patient Matters",
path: "contribution/?scrollTo=patient-matters"
},
{
name: "Wiki",
title: "Wiki",
path: "contribution/?scrollTo=wiki"
path: "contribution/?scrollTo=our-collaborations"
},
]
},
......
src/sidebars/conS.tsx
View file @ 3fd292fd
......@@ -11,9 +11,9 @@ export function ConSidebar(){
const tabs = [
{ tab: "BFH European MeetUp", subtabs: ["Aftervideo", "Timeline", "About", "Posters", "Guideline", "Gallery", "Downloads"]},
{tab: "Safety Guideline"},
{tab: "Collaborations"},
{tab: "Patient Matters"},
{tab: "Wiki"}
{tab: "Abstract"},
{ tab: "BFH European MeetUp", subtabs: ["Aftervideo", "Timeline", "About", "Posters", "Gallery", "Downloads"]},
{tab: "Our Collaborations", subtabs: ["MeetUp Guideline", "LNP Handbook"]},
{tab: "Our Contributions", subtabs: ["Biosafety & Security", "Wiki Developement"]},
{tab: "Conclusion"}
];
src/sidebars/engS.tsx
View file @ 3fd292fd
......@@ -9,9 +9,32 @@ export function EngSide(){
let nums = ["rep1", "rep2", "rep3", "rep4", "rep5", "rep6", "trf1", "trf2", "trf3", "trf4", "pe1", "pe2", "pe3", "peg1", "peg2", "peg3", "peg4", "peg5",
"nic1", "nic2", "nic3", "nic4", "nic5", "nic6","del1", "del2", "del3", "del4", "del5"]
useEffect(() => {
window.addEventListener('scroll', handleScroll);
return () => window.removeEventListener('scroll', handleScroll);
}, []);
window.addEventListener("scroll", handleScroll);
return () => window.removeEventListener("scroll", handleScroll);
}, [nums]);
// Restore active tab on reload
useEffect(() => {
const params = new URLSearchParams(window.location.search);
const tabId = params.get("tab");
const subTabId = params.get("subTab");
if (tabId) {
// Find the parent element and mark it as active
const parentTab = document.querySelector(`#parent-${tabId}`);
if (parentTab) {
document.querySelectorAll(".active-sideitem").forEach((el) => el.classList.remove("active-sideitem"));
parentTab.classList.add("active-sideitem");
}
// Optionally expand the subtab if available
if (subTabId) {
const subTab = document.querySelector(`#${subTabId}`);
if (subTab) {
subTab.classList.add("highlight-subitem"); // Add a custom class for highlighting subtabs if needed
}
}
}
}, []);
const handleScroll = () => {
for(let idx in nums){
/* console.log("We are looking at 'item' = " + nums[idx]) */
......
src/sidebars/igbS.tsx
View file @ 3fd292fd
......@@ -14,6 +14,5 @@ export function iGemBielefeldSidebar(){
const tabs = [
{tab: "Bielefeld University"},
{ tab: "History"},
{ tab: "Steering Committee", subtabs: ["Function", "Jörn"]},
{tab: "Future"}
{ tab: "Steering Committee"}
];
\ No newline at end of file
src/sidebars/jugS.tsx
View file @ 3fd292fd
......@@ -15,5 +15,6 @@ const tabs = [
{tab: "Best Integrated Human Practice"},
{tab: "Safety & Security"},
{tab: "Best New Basic Part"},
{tab: "Conclusion"}
{tab: "Conclusion"},
{tab: "Judging Session"}
];
src/sidebars/prtS.tsx
View file @ 3fd292fd
......@@ -11,8 +11,8 @@ export function PartSidebar(){
const tabs = [
{ tab: "Introduction", subtabs: ["Description", "Characterization"]},
{ tab: "Process", subtabs: ["EC", "Design and Build"]},
{tab: "Experiments", subtabs: ["Cloning", "Nikase-Assay", "Activity Experiments"]},
{tab: "Parts Collection", subtabs: ["Plasmids", "Basic Parts", "Composite Parts"]},
{ tab: "Description", subtabs: ["Introduction", "Prime Editor & pegRNA", "Conclusion"]},
{ tab: "Characterization", subtabs: ["Design & Functionality", "Adaptions"]},
{tab: "Experiments", subtabs: ["Cloning", "pegRNA Screening", "Nickase Assay"]},
{tab: "Parts Collection", subtabs: ["Basic Parts"]},
];
src/sidebars/resS.tsx
View file @ 3fd292fd
......@@ -13,7 +13,7 @@ export function ResultSidebar(){
}
const tabs = [
{ tab: "Abstract" , subtabs: ["Introduction", "Goals and Milestones"]},
{tab: "Experimental Design", subtabs: ["Proof of Concept", "PrimeGuide", "LNP Synthesis", "Cellculture", "Downstream Applications"]},
// {tab: ""},
{ tab: "Abstract"},
{tab: "Experimental Design", subtabs: ["Proof of Concept", "PrimeGuide", "LNP Synthesis", "Primary Cell Culture", "Downstream Applications", "PreCyse Results"]},
{tab: "Supplementary Material"}
];
\ No newline at end of file
src/sources/description-sources.tsx
View file @ 3fd292fd
......@@ -8,1236 +8,887 @@ export default function DescSources(){
)
}
const bibtexSources = [
`@article{article,
author={Rodrigues, Roberta and Gabetta, Carmen S. and Pedro, Karla P. and Valdetaro, Fabioand Fernandes, Maria I. M. and Magalhães, Patrícia K. R. and Januário, José N., Maciel, Léa M. Z.},
year={2008},
pages={475-484},
title={Cystic fibrosis and neonatal screening},
volume={24},
journal={Cadernos de Saúde Pública},
doi={10.1590/S0102-311X2008001600002}
}`,
`@article{article,
author={Farrell, Philip M. and Rock, Michael J. and Baker, Mei W.},
year={2020},
pages={401},
title={The Impact of the CFTR Gene Discovery on Cystic Fibrosis Diagnosis, Counseling, and Preventive Therapy},
volume={11(4)},
journal={Genes},
doi={10.3390/genes11040401}
}`,
`@article{article,
author={Lukacs, Gergely L. and Verkman, A. S.},
year={2012},
pages={81-91},
title={ CFTR: folding, misfolding and correcting the ΔF508 conformational defect},
volume={18(2)},
journal={Trends in molecular medicine},
doi={10.1016/j.molmed.2011.10.003}
}`,
`@misc{ECFS2024internet,
author={European Cystic Fibrosis Society},
title={2022 Highlight Report: Cystic Fibrosis in Europe, Facts and Figures},
year={2022},
url={https://www.ecfs.eu/sites/default/files/240619_PR_Highlights%20Report_page.pdf},
note={Zugriff am 27. September 2024}
}`,
`@article{article,
author={Bobadilla, J. L. and Macke Jr, M. and Fine, J. P. and Farrell, P. M.},
year={2002},
pages={575-606},
title={Cystic fibrosis: A worldwide analysis of CFTR mutations - correlation with incidence data and application to screening},
volume={19(6)},
journal={Human Mutation},
doi={10.1002/humu.10041}
}`,
`@article{10.3389/fphar.2019.01662,
author={Lopes-Pacheco, Miquéias},
title={CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine},
journal={Frontiers in Pharmacology},
volume={10},
year={2020},
doi={10.3389/fphar.2019.01662},
url={https://www.frontiersin.org/articles/10.3389/fphar.2019.01662}
}`,
`@misc{arena2024internet,
author={Clinical Trials Arena},
title={Cystic fibrosis: global clinical trials landscape and treatments},
year={2024},
url={https://www.clinicaltrialsarena.com/sponsored/cystic-fibrosis-global-clinical-trials-landscape-and-treatments/},
note={Zugriff am 23. September 2024}
}`,
`@misc{MSDmanual2024internet,
author={Sawicki, G},
title={Mukoviszidose (zystische Fibrose, CF)},
year={2023},
url={https://www.msdmanuals.com/de-de/heim/gesundheitsprobleme-von-kindern/mukoviszidose/mukoviszidose-zystische-fibrose-cf},
note={Zugriff am 23. September 2024}
}`,
`@misc{patient2024internet,
author={Burkhart, M. and Nährlich, L.},
title={Zahlen, Daten und Fakten für Patienten und Angehörige},
year={2022},
url={https://www.muko.info/fileadmin/user_upload/was_wir_tun/register/berichtsbaende/patientenberichtsband.pdf},
note={Zugriff am 23. September 2024}
}`,
`@misc{Muko82024internet,
author={Mukoviszidose e.V},
title={Was ist Mukoviszidose/ Cystische Fibrose (CF)?},
year={2024},
url={https://www.muko.info/mukoviszidose/ueber-die-erkrankung},
note={Zugriff am 23. September 2024}
}`,
`@article{Elborn2016,
title={Cystic fibrosis},
volume={388},
ISSN={0140-6736},
url={http://dx.doi.org/10.1016/s0140-6736(16)00576-6},
DOI={10.1016/s0140-6736(16)00576-6},
number={10059},
journal={The Lancet},
publisher={Elsevier BV},
author={Elborn, J Stuart},
year={2016},
month={nov},
pages={2519-2531}
}`,
`@book{2001,
title={Cystische Fibrose},
author={Reinhardt, D. and Götz, M. and Kraemer, R. and Schöni, M.},
ISBN={9783642567964},
url={http://dx.doi.org/10.1007/978-3-642-56796-4},
DOI={10.1007/978-3-642-56796-4},
publisher={Springer Berlin Heidelberg},
year={2001}
}`,
`@article{biom4020498,
author={Wang, Xiaodong Robert and Li, Chenglong},
title={Decoding F508del Misfolding in Cystic Fibrosis},
journal={Biomolecules},
volume={4},
year={2014},
number={2},
pages={498--509},
url={https://www.mdpi.com/2218-273X/4/2/498},
PubMedID={24970227},
ISSN={2218-273X},
DOI={10.3390/biom4020498}
}`,
/* 14 */
`@article{PUCHELLE2002115,
title={Airway mucus in cystic fibrosis},
journal={Paediatric Respiratory Reviews},
volume={3},
number={2},
pages={115-119},
year={2002},
issn={1526-0542},
doi={https://doi.org/10.1016/S1526-0550(02)00005-7},
url={https://www.sciencedirect.com/science/article/pii/S1526055002000057},
author={Puchelle, Edith and Bajolet, Odile and Abély, Michel},
keywords={cystic fibrosis, airway mucus, airway surface liquid, rheology, mucociliary clearance, mucus cough transport}
}`, /* 15 */
`@article{Anzalone2019,
author = {Anzalone, Andrew and Randolph, Peyton and Davis, Jessie and Sousa, Ayalur and Koblan, Luke and Levy, Jonathan and Chen, Patrick and Wilson, Charlotte and Newby, Greg and Raguram, Aditya and Liu, David},
title = {Search-and-replace genome editing without double-strand breaks or donor DNA},
journal = {Nature},
volume = {576},
pages = {149--157},
year = {2019},
doi = {10.1038/s41586-019-1711-4}
`
@article{Rodrigues_2008,
title = {Cystic fibrosis and neonatal screening},
author = {
Rodrigues, Roberta and Gabetta, Carmen S. and Pedro, Karla P. and Valdetaro,
Fabio and Fernandes, Maria I. M. and Magalhães, Patrícia K. R. and Januário,
José N. and Maciel, Léa M. Z.
},
year = 2008,
journal = {Cadernos de Saúde Pública},
publisher = {SciELO},
volume = 24,
number = {suppl 4},
pages = {s592--s602},
doi = {10.1590/S0102-311X2008001600002},
url = {https://doi.org/10.1590/S0102-311X2008001600002}
}`,`
@article{genes11040401,
title = {
The Impact of the CFTR Gene Discovery on Cystic Fibrosis Diagnosis,
Counseling, and Preventive Therapy
},
author = {Farrell, Philip M. and Rock, Michael J. and Baker, Mei W.},
year = 2020,
journal = {Genes},
volume = 11,
number = 4,
doi = {10.3390/genes11040401},
issn = {2073-4425},
url = {https://www.mdpi.com/2073-4425/11/4/401},
article-number = 401,
pubmedid = 32276344
}`,`
@article{zgzugzuzk,
title = {
CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision
Medicine
},
author = {Lopes-Pacheco, Miquéias},
year = 2020,
journal = {Frontiers in Pharmacology},
volume = 10,
doi = {10.3389/fphar.2019.01662},
issn = {1663-9812},
url = {
https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2019.01662
}
}`,`
@misc{clinicaltrialsarena2024,
title = {Cystic fibrosis: global clinic trials landscape and treatments},
author = {Novotech},
year = 2024,
url = {
https://www.clinicaltrialsarena.com/sponsored/cystic-fibrosis-global-clinical-trials-landscape-and-treatments/
},
note = {Zugriff am 23. September 2024}
}`,`
@misc{MSDmanual2024internet,
title = {Mukoviszidose (zystische Fibrose, CF)},
author = {Gregory Sawicki},
year = 2023,
url = {
https://www.msdmanuals.com/de-de/heim/gesundheitsprobleme-von-kindern/mukoviszidose/mukoviszidose-zystische-fibrose-cf​
},
note = {Zugriff am 23. September 2024}
}`,`
@misc{patient2024internet,
title = {Zahlen, Daten und Fakten für Patienten und Angehörige},
author = {M. Burkhart, L. Nährlich},
year = 2022,
url = {
https://www.muko.info/fileadmin/user_upload/was_wir_tun/register/berichtsbaende/patientenberichtsband.pdf​
},
note = {Zugriff am 23. September 2024}
}`,`
@book{Baird2022,
title = {
Dorothy Hansine Andersen: The Life and Times of the Pioneering
Physician-Scientist Who Identified Cystic Fibrosis
},
author = {Baird, John Scott},
year = 2022,
publisher = {Springer International Publishing},
doi = {10.1007/978-3-030-87484-1},
isbn = 9783030874841,
url = {http://dx.doi.org/10.1007/978-3-030-87484-1}
}`,`
@book{Ballmann2008-bt,
title = {CF-Manual},
author = {Ballmann, Manfred and Smaczny, Christina},
year = 2008,
language = {de}
}`,`
@article{article,
title = {CFTR: folding, misfolding and correcting the ΔF508 conformational defect},
author = {Lukacs, Gergely L.; Verkman, A. S.},
year = 2012,
month = 2,
journal = {Trends in molecular medicine},
volume = {{18(2)}},
pages = {81--91},
doi = {10.1016/j.molmed.2011.10.003}
}`,`
@misc{muko1internet2024,
title = {Was ist Mukoviszidose/ Cystische Fibrose (CF)?},
author = {Mukoviszidose e.V.},
year = 2024,
url = {https://www.muko.info/mukoviszidose/ueber-die-erkrankung​},
note = {Zugriff am 23. September 2024}
}`,`
@misc{muko2internet2024,
title = {Was ist Mukoviszidose/ Cystische Fibrose (CF)?},
author = {Mukoviszidose e.V.},
year = 2024,
url = {https://www.muko.info/mukoviszidose/ueber-die-erkrankung/ursache​},
note = {Zugriff am 23. September 2024}
}`,`
@article{CFTRchannel2013,
title = {The CFTR ion channel: gating, regulation, and anion permeation},
author = {Hwang, Tzyh-Chang and Kirk, Kevin L.},
year = 2013,
journal = {Cold Spring Harbor Perspectives in Medicine},
publisher = {Cold Spring Harbor Laboratory Press},
volume = 3,
number = 1,
pages = {a009498},
doi = {10.1101/cshperspect.a009498}
}`,`
@article{CFTRfunction2008,
title = {CFTR Function and Prospects for Therapy},
author = {Riordan, John R.},
year = 2008,
journal = {Annual Review of Biochemistry},
publisher = {Annual Reviews},
volume = 77,
number = 1,
pages = {701--26},
doi = {10.1146/annurev.biochem.75.103004.142532}
}`,`
@article{ABCprotein2006,
title = {
The ABC protein turned chloride channel whose failure causes cystic fibrosis
},
author = {Gadsby, D.; Vergani, P. & Csanády, L.},
year = 2006,
journal = {Nature},
publisher = {Nature},
volume = 440,
number = 1,
pages = {477--483},
doi = {https://doi.org/10.1038/nature04712}
}`,`
@article{Sheppard1999,
title = {Structure and function of the CFTR chloride channel},
author = {Sheppard, David N. and Welsh, Michael J.},
year = 1999,
journal = {Physiological Reviews},
publisher = {American Physiological Society},
volume = 79,
number = {1 Suppl},
pages = {S23--S45},
doi = {10.1152/physrev.1999.79.1.S23}
}
`,/* 16 */
`@article{Doman2023,
author = {Doman, Jacob and Pandey, Siddharth and Neugebauer, Maria and An, Meiqi and Davis, Jessie and Randolph, Peyton and McElroy, Andrew and Gao, Xiaoxu and Raguram, Aditya and Richter, Markus and Everette, Keaton and Banskota, Saroj and Tian, Kangrui and Tao, Yuhang and Tolar, Jakub and Osborn, Matthew and Liu, David},
title = {Phage-assisted evolution and protein engineering yield compact, efficient prime editors},
journal = {Cell},
volume = {186},
pages = {3983--4002.e26},
year = {2023},
doi = {10.1016/j.cell.2023.07.039}
`,`
@article{article,
title = {
Unravelling the Regions of Mutant F508del-CFTR More Susceptible to the Action
of Four Cystic Fibrosis Correctors
},
author = {Amico, Giulia; Brandas, Chiara; Moran, Oscar; Baroni, Debora},
year = 2019,
month = 1,
journal = {International Journal of Molecular Sciences},
volume = {{20(21)}},
pages = 5463,
doi = {10.3390/ijms20215463}
}`,`
@misc{cff2024digestive,
title = {Managing CF: Digestive Tract},
author = {Cystic Fibrosis Foundation},
year = 2024,
url = {https://www.cff.org/managing-cf/digestive-tract},
note = {Zugriff am 2. Dezember 2024}
}`,`
@misc{mukoleitlinien2024internet,
title = {
Mukoviszidose (Cystische Fibrose): Ernährung und exokrine
Pankreasinsuffizienz
},
author = {M. Stern, H. Ellemunter, B. Palm, H.-G. Posselt, C. Smaczny},
year = 2011,
url = {
https://www.muko.info/fileadmin/user_upload/was_wir_tun/leitlinien/LL_S1_mukoviszidose_ernaehrung_exokrine_pankreasinsuffizienz.pdf​
},
note = {Zugriff am 25. September 2024}
}`,`
@misc{CFF42024internet,
title = {The Digestive Tract},
author = {Cystic Fibrosis Foundation},
year = 2024,
url = {https://www.cff.org/managing-cf/digestive-tract​},
note = {Zugriff am 25. September 2024}
}`,`
@misc{muko2024therapie,
title = {Therapie der Mukoviszidose},
author = {Mukoviszidose e.V.},
year = 2024,
url = {https://www.muko.info/mukoviszidose/therapie-der-mukoviszidose#c1571},
note = {Zugriff am 2. Dezember 2024}
}
`,`
@misc{ncbi2024cf,
title = {Cystic Fibrosis: The Basics},
author = {National Center for Biotechnology Information (NCBI)},
year = 2024,
url = {https://www.ncbi.nlm.nih.gov/books/NBK279303/},
note = {Zugriff am 2. Dezember 2024}
}`,`
@article{DeLisle2013,
title = {The Cystic Fibrosis Intestine},
author = {De Lisle, R. C. and Borowitz, D.},
year = 2013,
month = jun,
journal = {Cold Spring Harbor Perspectives in Medicine},
publisher = {Cold Spring Harbor Laboratory},
volume = 3,
number = 9,
pages = {a009753-a009753},
doi = {10.1101/cshperspect.a009753},
issn = {2157-1422},
url = {http://dx.doi.org/10.1101/cshperspect.a009753}
}`,`
@misc{gesundheit2024leber,
title = {Die Funktionen der Leber},
author = {Gesundheit.gv.at},
year = 2024,
url = {https://www.gesundheit.gv.at/krankheiten/leber/funktion.html},
note = {Zugriff am 2. Dezember 2024}
}`,`
@misc{cfuk2024body,
title = {How Does Cystic Fibrosis Affect the Body?},
author = {Cystic Fibrosis Trust},
year = 2024,
url = {
https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/how-does-cystic-fibrosis-affect-the-body
},
note = {Zugriff am 2. Dezember 2024}
}`,`
@misc{ncbi2024glossary,
title = {Glossary of Gene Therapy Terms},
author = {National Center for Biotechnology Information (NCBI)},
year = 2024,
url = {https://www.ncbi.nlm.nih.gov/books/NBK556086/},
note = {Zugriff am 2. Dezember 2024}
}`,`
@misc{CFT2024internet,
title = {Fertility and cystic fibrosis},
author = {Cystic Fibrosis Trust},
year = 2024,
url = {
https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/how-does-cystic-fibrosis-affect-the-body/symptoms-of-cystic-fibrosis/fertility
​
},
note = {Zugriff am 25. September 2024}
}`,`
@misc{CFSource2024internet,
title = {Abnehmende Lungenfunktion als Leitsymptom bei CF},
author = {CF Source},
year = 2023,
url = {http://www.cfsource.at/de/cystische-fibrose/symptome/lunge​},
note = {Zugriff am 25. September 2024}
}`,`
@article{Rowe2005,
title = {Cystic Fibrosis},
author = {Rowe, Steven M. and Miller, Stacey and Sorscher, Eric J.},
year = 2005,
month = may,
journal = {New England Journal of Medicine},
publisher = {Massachusetts Medical Society},
volume = 352,
number = 19,
pages = {1992-2001},
doi = {10.1056/nejmra043184},
issn = {1533-4406},
url = {http://dx.doi.org/10.1056/NEJMra043184}
}`,`
@article{OSULLIVAN20091891,
title = {Cystic fibrosis},
author = {O'Sullivan, Brian and Freedman, Steven},
year = 2009,
journal = {The Lancet},
volume = 373,
number = 9678,
pages = {1891--1904},
doi = {https://doi.org/10.1016/S0140-6736(09)60327-5},
issn = {0140-6736},
url = {https://www.sciencedirect.com/science/article/pii/S0140673609603275}
}`,`
@misc{Muko62024internet,
title = {Mukoviszidose: Medikamente und Behandlung},
author = {Lungen Informationsdienst},
year = 2023,
url = {https://www.lungeninformationsdienst.de/krankheiten/mukoviszidose/therapie​},
note = {Zugriff am 25. September 2024}
}`,`
@book{Lambrechts_2023,
title = {Musculoskeletal Abnormalities Caused by Cystic Fibrosis},
author = {Lambrechts, Mark},
year = 2023,
month = mar,
booktitle = {Advances in Skeletal Muscle Health and Disease},
publisher = {IntechOpen},
doi = {10.5772/intechopen.104591},
isbn = 9781803562735,
issn = {2631-8261},
url = {http://dx.doi.org/10.5772/intechopen.104591}
}`,`
@misc{Anatomy2024internet,
title = {Anatomy, Skin (Integument), Epidermis},
author = {Yousef, H., Alhajj, M., Fakoya AO, et al.},
year = 2024,
url = {https://www.ncbi.nlm.nih.gov/books/NBK470464/},
note = {Zugriff am 25. September2024}
}`,`
@article{Wenk_2010,
title = {Cystic Fibrosis Presenting With Dermatitis},
author = {Wenk, Kurt S. and Higgins, Kristen B. and Greer, Kenneth E.},
year = 2010,
month = feb,
journal = {Archives of Dermatology},
publisher = {American Medical Association (AMA)},
volume = 146,
number = 2,
doi = {10.1001/archdermatol.2009.361},
issn = {0003-987X},
url = {http://dx.doi.org/10.1001/archdermatol.2009.361}
}`,`
@article{DAE193172,
title = {Cystic Fibrosis},
author = {Naehrig, Susanne and Chao, Cho-Ming and Naehrlich, Lutz},
year = 2017,
journal = {Dtsch Arztebl International},
volume = 114,
number = {33-34},
pages = {564--574},
doi = {10.3238/arztebl.2017.0564},
url = {https://www.aerzteblatt.de/int/article.asp?id=193172},
eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
}`,`
@article{Guta2021,
title = {
Global Burden of Anxiety and Depression among Cystic Fibrosis Patient:
Systematic Review and Meta-Analysis
},
author = {
Guta, Mistire Teshome and Tekalign, Tiwabwork and Awoke, Nefsu and Fite,
Robera Olana and Dendir, Getahun and Lenjebo, Tsegaye Lolaso
},
year = 2021,
month = jul,
journal = {International Journal of Chronic Diseases},
publisher = {Hindawi Limited},
volume = 2021,
pages = {1-9},
doi = {10.1155/2021/6708865},
issn = {2356-6981},
url = {http://dx.doi.org/10.1155/2021/6708865},
editor = {Rao, Kosagi S. Jagannatha}
}`,`
@book{Behrhorst2020,
title = {Mental Health in Cystic Fibrosis},
author = {Behrhorst, Kathryn L. and Everhart, Robin S. and Schechter, Michael S.},
year = 2020,
booktitle = {Cystic Fibrosis},
publisher = {Springer International Publishing},
pages = {429-447},
doi = {10.1007/978-3-030-42382-7_21},
isbn = 9783030423827,
issn = {2197-7380},
url = {http://dx.doi.org/10.1007/978-3-030-42382-7_21}
}`,`
@article{Southern01,
title = {Newborn screening for cystic fibrosis},
author = {Southern, KW, Mérelle, MME, Dankert‐Roelse, JE, and Nagelkerke, A},
year = 2009,
journal = {Cochrane Database of Systematic Reviews},
publisher = {John Wiley & Sons, Ltd},
number = 1,
doi = {10.1002/14651858.CD001402.pub2},
issn = {1465-1858},
url = {https://doi.org//10.1002/14651858.CD001402.pub2},
keywords = {
*Neonatal Screening; Cystic Fibrosis [*diagnosis]; Humans; Infant, Newborn;
Quality of Life; Randomized Controlled Trials as Topic; Survival Analysis
}
}`,`
@book{Paracchini2012,
title = {
Cystic Fibrosis Newborn Screening: Distribution of Blood Immunoreactive
Trypsinogen Concentrations in Hypertrypsinemic Neonates
},
author = {
Paracchini, Valentina and Seia, Manuela and Raimondi, Sara and Costantino,
Lucy and Capasso, Patrizia and Porcaro, Luigi and Colombo, Carla and
Coviello, Domenico A. and Mariani, Tiziana and Manzoni, Emanuela and
Sangiovanni, Monica and Corbetta, Carlo
},
year = 2012,
booktitle = {JIMD Reports - Case and Research Reports, 2012/1},
publisher = {Springer Berlin Heidelberg},
address = {Berlin, Heidelberg},
pages = {17--23},
doi = {10.1007/8904_2011_55},
isbn = {978-3-642-25752-0},
url = {https://doi.org/10.1007/8904_2011_55},
abstract = {
The IRT screening test for the use in diagnosing newborns with CF has a high
sensitivity but is not very specific resulting in a large number of screened
positive infants found to have a normal sweat test. The aim of this study was
to analyze the differences in b-IRT levels among different groups of newborns
positive to NBS.
}
}`,`
@article{Barben2006,
title = {A survey of newborn screening for cystic fibrosis in Europe},
author = {
Barben, Jürg and Southern, Kevin W. and Greaves, David A. and Heaf, Annick and
Dodge, Michael J.
},
year = 2006,
journal = {Journal of Cystic Fibrosis},
volume = 5,
number = 3,
pages = {143--148},
doi = {10.1016/j.jcf.2006.05.008},
url = {
https://www.cysticfibrosisjournal.com/article/S1569-1993(06)00073-7/fulltext
}
}`,`
@misc{cff2024newborn,
title = {Newborn Screening for CF},
author = {Cystic Fibrosis Foundation},
year = 2024,
url = {https://www.cff.org/intro-cf/newborn-screening-cf},
note = {Zugriff am 2. Dezember 2024}
}
`
,
`@article{Gadsby2006,
title={The ABC protein turned chloride channel whose failure causes cystic fibrosis},
author={Gadsby, David C. and Vergani, Paola and Csanády, L{\'a}szl{\'o}},
journal={Nature},
volume={440},
number={7083},
pages={477--483},
year={2006},
publisher={Nature Publishing Group},
doi={10.1038/nature04712}
} `,
`@article{Hwang2013,
title={The CFTR ion channel: gating, regulation, and anion permeation},
author={Hwang, Tzyh-Chang and Kirk, Kevin L.},
journal={Cold Spring Harbor Perspectives in Medicine},
volume={3},
number={1},
pages={a009498},
year={2013},
publisher={Cold Spring Harbor Laboratory Press},
doi={10.1101/cshperspect.a009498}
} `,
`@article{Riordan2008,
title={CFTR function and prospects for therapy},
author={Riordan, John R.},
journal={Annual Review of Biochemistry},
volume={77},
pages={701--726},
year={2008},
publisher={Annual Reviews},
doi={10.1146/annurev.biochem.75.103004.142532}
} `,
`@article{Sheppard1999,
title={Structure and function of the CFTR chloride channel},
author={Sheppard, David N. and Welsh, Michael J.},
journal={Physiological Reviews},
volume={79},
number={1 Suppl},
pages={S23--S45},
year={1999},
publisher={American Physiological Society},
doi={10.1152/physrev.1999.79.1.S23}
} `,
` @article{Chen2008,
title={CFTR activation by phosphorylation},
author={Chen, Jeng-Haur and Chang, Xue-Bao},
journal={Journal of Biomedical Science},
volume={15},
number={6},
pages={659--672},
year={2008},
publisher={Springer},
doi={10.1007/s11373-008-9260-4}
} `,
`@article{article,
author={Lukacs, Gergely L.; Verkman, A. S.},
year={2012},
month={2},
pages={81-91},
title={ CFTR: folding, misfolding and correcting the ΔF508 conformational defect},
volume={18(2)},
journal={Trends in molecular medicine},
doi={10.1016/j.molmed.2011.10.003}
} `,
`@article{article,
author={Farrell, Philip M.; Rock, Michael J.; Baker, Mei W.},
year={2020},
month={04},
pages={401},
title={The Impact of the CFTR Gene Discovery on Cystic Fibrosis Diagnosis, Counseling, and Preventive Therapy},
volume={11(4)},
journal={Genes},
doi={10.3390/genes11040401}
} `,
`@article{article,
author={Rodrigues, Roberta; Gabetta, Carmen S.; Pedro, Karla P.; Valdetaro, Fabio; Fernandes, Maria I. M.; Magalhães, Patrícia K. R.; Januário, José N., Maciel, Léa M. Z.},
year={2008},
month={ },
pages={475-484},
title={Cystic fibrosis and neonatal screening},
volume={24},
journal={Cadernos de Saúde Pública},
doi={10.1590/S0102-311X2008001600002}
} `,
`@article{article,
author={Amico, Giulia; Brandas, Chiara; Moran, Oscar; Baroni, Debora},
year={2019},
month={1},
pages={5463},
title={Unravelling the Regions of Mutant F508del-CFTR More Susceptible to the Action of Four Cystic Fibrosis Correctors},
volume={20(21)},
journal={International Journal of Molecular Sciences},
doi={10.3390/ijms20215463}
} `,
`@book{Braun2022Basislehrbuch,
isbn = {9783437411441},
language = {de},
numpages = {1208},
title = {Basislehrbuch Innere Medizin},
url = {https://books.google.com/books/about/Basislehrbuch_Innere_Medizin.html?hl=&id=BS8tzwEACAAJ},
author = {Braun, Jörg and Müller-Wieland, Dirk},
date = {2022-09-20},
year = {2022},
month = {9},
day = {20},
} `,
/* 27 */
`@article{Wenk_2010,
title={Cystic Fibrosis Presenting With Dermatitis},
volume={146},
ISSN={0003-987X},
url={http://dx.doi.org/10.1001/archdermatol.2009.361},
DOI={10.1001/archdermatol.2009.361},
number={2},
journal={Archives of Dermatology},
publisher={American Medical Association (AMA)},
author={Wenk, Kurt S. and Higgins, Kristen B. and Greer, Kenneth E.},
year={2010},
month={feb}
}`,
/* 28 */
`@misc{mukoleitlinien2024internet,
author = {M. Stern, H. Ellemunter, B. Palm, H.-G. Posselt, C. Smaczny},
title = {Mukoviszidose (Cystische Fibrose): Ernährung und exokrine Pankreasinsuffizienz},
year = {2011},
url = {https://www.muko.info/fileadmin/user_upload/was_wir_tun/leitlinien/LL_S1_mukoviszidose_ernaehrung_exokrine_pankreasinsuffizienz.pdf​},
note = {Zugriff am 25. September 2024}
} `,
`
@misc{CFF42024internet,
author = {Cystic Fibrosis Foundation},
title = {The Digestive Tract},
year = {2024},
url = {https://www.cff.org/managing-cf/digestive-tract​},
note = {Zugriff am 25. September 2024}
} `,
`
@article{Pozo2020,
title = {Cystic fibrosis-related diabetes: The unmet need},
volume = {11},
ISSN = {1948-9358},
url = {http://dx.doi.org/10.4239/wjd.v11.i6.213},
DOI = {10.4239/wjd.v11.i6.213},
number = {6},
journal = {World Journal of Diabetes},
publisher = {Baishideng Publishing Group Inc.},
author = {Pozo, Leonardo and Bello, Fatimah and Mendez, Yamely and Surani, Salim},
year = {2020},
month = jun,
pages = {213–217}
} `,
`@misc{Muko72024internet,
author = {Mukoviszidose e.V.},
title = {Therapie der Mukoviszidose},
year = {2017},
url = {https://www.muko.info/mukoviszidose/therapie-der-mukoviszidose#c1571​},
note = {Zugriff am 25. September 2024}
} `,
`@misc{intestine2024internet,
author = {Institute for Quality and Efficiency in Health Care (IQWiG)},
title = {In brief: How does the intestine work?},
year = {2021},
url = {https://www.ncbi.nlm.nih.gov/books/NBK279303​},
note = {Zugriff am 25. September 2024}
} `,
`@article{DeLisle2013,
title = {The Cystic Fibrosis Intestine},
volume = {3},
ISSN = {2157-1422},
url = {http://dx.doi.org/10.1101/cshperspect.a009753},
DOI = {10.1101/cshperspect.a009753},
number = {9},
journal = {Cold Spring Harbor Perspectives in Medicine},
publisher = {Cold Spring Harbor Laboratory},
author = {De Lisle, R. C. and Borowitz, D.},
year = {2013},
month = jun,
pages = {a009753–a009753}
} `,
`@misc{Gesund2024internet,
author = {GESUNDheit.GV.AT},
title = {Leber: Aufbau und Funktion},
year = {2024},
url = {https://www.gesundheit.gv.at/krankheiten/leber/funktion.html​},
note = {Zugriff am 25. September 2024}
} `,
`@misc{CFT22024internet,
author = {Cystic Fibrosis Trust},
title = {How does cystic fibrosis affect the body?},
year = {2024},
url = {https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/how-does-cystic-fibrosis-affect-the-body​},
note = {Zugriff am 25. September 2024}
} `,
`@misc{Liver2024internet,
author = {Betapudi, B., Aleem, A., Kothadia, JP.},
title = {Cystic Fibrosis and Liver Disease},
year = {2024},
url = {https://www.ncbi.nlm.nih.gov/books/NBK556086/​},
note = {Zugriff am 25. September 2024}
} `,
`@book{netter2018atlas,
title={Atlas of Human Anatomy},
author={Netter, Frank H.},
year={2018},
edition={7th},
publisher={Elsevier}
} `,
`@book{tortora2016principles,
title={Principles of Anatomy and Physiology},
author={Tortora, Gerard J. and Derrickson, Bryan H.},
year={2016},
edition={15th},
publisher={Wiley}
} `,
`@article{Dodge_1995,
title={Male fertility in cystic fibrosis},
volume={346},
ISSN={0140-6736},
url={http://dx.doi.org/10.1016/s0140-6736(95)91431-5},
DOI={10.1016/s0140-6736(95)91431-5},
number={8975},
journal={The Lancet},
publisher={Elsevier BV},
author={Dodge, J.A.},
year={1995},
month=sep,
pages={587–588}
} `,
`@article{Davis_2006, title={Cystic Fibrosis Since 1938}, volume={173}, ISSN={1535-4970}, url={http://dx.doi.org/10.1164/rccm.200505-840oe}, DOI={10.1164/rccm.200505-840oe}, number={5}, journal={American Journal of Respiratory and Critical Care Medicine}, publisher={American Thoracic Society}, author={Davis, Pamela B.}, year={2006}, month=mar, pages={475–482} } `,
`@misc{CFT2024internet,
author = {Cystic Fibrosis Trust},
title = {Fertility and cystic fibrosis},
year = {2024},
url = {https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/how-does-cystic-fibrosis-affect-the-body/symptoms-of-cystic-fibrosis/fertility ​},
note = {Zugriff am 25. September 2024}
} `,
`@misc{SGW2024internet,
author = {Stiftung Gesundheit Wissen},
title = {Wie funktioniert die Lunge?},
year = {2023},
url = {https://www.stiftung-gesundheitswissen.de/gesundes-leben/koerper-wissen/wie-funktioniert-die-lunge​},
note = {Zugriff am 25. September 2024}
} `,
`@misc{CFSource2024internet,
author = {CF Source},
title = {Abnehmende Lungenfunktion als Leitsymptom bei CF},
year = {2023},
url = {http://www.cfsource.at/de/cystische-fibrose/symptome/lunge​},
note = {Zugriff am 25. September 2024}
} `,
`@misc{CFF32024internet,
author = {Cystic Fibrosis Foundation},
title = {About Cystic Fibrosis},
year = {2024},
url = {https://www.cff.org/intro-cf/about-cystic-fibrosis​},
note = {Zugriff am 25. September 2024}
} `,
`@article{Rowe2005,
title = {Cystic Fibrosis},
volume = {352},
ISSN = {1533-4406},
url = {http://dx.doi.org/10.1056/NEJMra043184},
DOI = {10.1056/nejmra043184},
number = {19},
journal = {New England Journal of Medicine},
publisher = {Massachusetts Medical Society},
author = {Rowe, Steven M. and Miller, Stacey and Sorscher, Eric J.},
year = {2005},
month = may,
pages = {1992–2001}
} `,
`@article{O_Sullivan_2009, title={Cystic fibrosis}, volume={373}, ISSN={0140-6736}, url={http://dx.doi.org/10.1016/s0140-6736(09)60327-5}, DOI={10.1016/s0140-6736(09)60327-5}, number={9678}, journal={The Lancet}, publisher={Elsevier BV}, author={O’Sullivan, Brian P and Freedman, Steven D}, year={2009}, month=may, pages={1891–1904} } `,
`@misc{Muko62024internet,
author = {Lungen Informationsdienst},
title = {Mukoviszidose: Medikamente und Behandlung},
year = {2023},
url = {https://www.lungeninformationsdienst.de/krankheiten/mukoviszidose/therapie​},
note = {Zugriff am 25. September 2024}
} `,
`@article{Dahdul_2012, title={A Unified Anatomy Ontology of the Vertebrate Skeletal System}, volume={7}, ISSN={1932-6203}, url={http://dx.doi.org/10.1371/journal.pone.0051070}, DOI={10.1371/journal.pone.0051070}, number={12}, journal={PLoS ONE}, publisher={Public Library of Science (PLoS)}, author={Dahdul, Wasila M. and Balhoff, James P. and Blackburn, David C. and Diehl, Alexander D. and Haendel, Melissa A. and Hall, Brian K. and Lapp, Hilmar and Lundberg, John G. and Mungall, Christopher J. and Ringwald, Martin and Segerdell, Erik and Van Slyke, Ceri E. and Vickaryous, Matthew K. and Westerfield, Monte and Mabee, Paula M.}, editor={Robinson-Rechavi, Marc}, year={2012}, month=dec, pages={e51070} } `,
`@book{Lambrechts_2023, title={Musculoskeletal Abnormalities Caused by Cystic Fibrosis}, ISBN={9781803562735}, ISSN={2631-8261}, url={http://dx.doi.org/10.5772/intechopen.104591}, DOI={10.5772/intechopen.104591}, booktitle={Advances in Skeletal Muscle Health and Disease}, publisher={IntechOpen}, author={Lambrechts, Mark}, year={2023}, month=mar } `,
`@misc{Anatomy2024internet,
author = {Yousef, H., Alhajj, M., Fakoya AO, et al.},
title = {Anatomy, Skin (Integument), Epidermis},
year = {2024},
url = {https://www.ncbi.nlm.nih.gov/books/NBK470464/},
note = {Zugriff am 25. September2024}
} `,
`@article{DAE193172,
author = {Naehrig, Susanne and Chao, Cho-Ming and Naehrlich, Lutz},
title = {{Cystic Fibrosis}},
journal = {Deutsches Ärzteblatt International},
volume = {114},
number = {33-34},
pages = {564-574},
doi = {10.3238/arztebl.2017.0564},
year = {2017},
abstract = {Background: Universal screening of newborn babies for cystic fibrosis was launched in Germany on 1 September 2016. Here we present up-to-date information on the diagnosis, treatment, and prognosis of this disease.Methods: This article is based on relevant publications retrieved by a selective search in PubMed, along with guidelines from Germany and abroad and systematic reviews.Results: Cystic fibrosis is caused by a gene mutation leading to dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It affects multiple organ systems—the lungs, pancreas, upper airways, liver, intestine, and reproductive organs—to varying degrees. Its incidence among newborn babies in Germany is between 1 in 3300 and 1 in 4800. Its diagnosis requires both clinical evidence (positive newborn screening, sibling[s] with cystic fibrosis, clinical signs) and the demonstration of CFTR dysfunction by an elevated chloride concentration in sweat, and/or two disease-causing mutations, and/or abnormal electrophysiological findings (nasal potential difference measurement, intestinal short-circuit current measurement). Patients should be cared for by specialized cystic fibrosis centers in close cooperation with their primary care physicians. The median life span of patients with this disease has risen steadily to the current value of 40 years. Aside from symptomatic treatment, the first mutation-specific treatments have recently become available.Conclusion: Early diagnosis and optimized treatment prolong the lives of persons with cystic fibrosis and improve their quality of life. Causally directed treatment for all patients and their effects on the course of disease are now central issues for further research.},
URL = {https://www.aerzteblatt.de/int/article.asp?id=193172},
eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
} `,
/* 53 */
`@book{sobotta_anatomie,
title={Atlas der Anatomie Band 3},
author={Sobotta, Johannes and Paulsen, Friedrich and Waschke, Jens},
year={2017},
publisher={Urban \& Fischer},
edition={24. Auflage},
isbn={9783437098185}
}`,
`@misc{Resp2024internet,
author={The Respiratory System},
title={Nase: Definition, Anatomie und Funktion},
year={2017},
url={https://www.therespiratorysystem.com/de/nose/},
note={Zugriff am 25. September 2024}
}`,
`@article{Guta2021,
title={Global Burden of Anxiety and Depression among Cystic Fibrosis Patient: Systematic Review and Meta-Analysis},
volume={2021},
ISSN={2356-6981},
url={http://dx.doi.org/10.1155/2021/6708865},
DOI={10.1155/2021/6708865},
journal={International Journal of Chronic Diseases},
publisher={Hindawi Limited},
author={Guta, Mistire Teshome and Tekalign, Tiwabwork and Awoke, Nefsu and Fite, Robera Olana and Dendir, Getahun and Lenjebo, Tsegaye Lolaso},
editor={Rao, Kosagi S. Jagannatha},
year={2021},
month={jul},
pages={1-9}
}`,
/* 56 */
`@article{Farrell2017,
title={Diagnosis of Cystic Fibrosis: Consensus Guidelines from the Cystic Fibrosis Foundation},
author={Farrell, Philip M. and White, Thomas B. and Ren, Carol L. and Hempstead, Sarah E. and Accurso, Frank and Derichs, Nico and Howenstine, Margaret S. and McColley, Susanna A. and Rock, Michael J. and Rosenfeld, Margaret and Campbell, Peter W.},
journal={Journal of Pediatrics},
volume={181S},
pages={4--15},
year={2017},
publisher={Elsevier},
doi={10.1016/j.jpeds.2016.09.064}
}`,
`@article{Castellani2008,
title={Consensus on the use and interpretation of cystic fibrosis mutation analysis in clinical practice},
author={Castellani, Carlo and Cuppens, H. and Macek Jr, Milan and Cassiman, Jean-Marie and Kerem, Eitan and Durie, Peter and Tullis, Elizabeth and Assael, B. M. and Bombieri, Cristina and Brown, Amanda and others},
journal={Journal of Cystic Fibrosis},
volume={7},
number={3},
pages={179--196},
year={2008},
publisher={Elsevier},
doi={10.1016/j.jcf.2008.03.009}
}`,
`@article{Comeau2004,
title={Population-based newborn screening for genetic disorders: the Massachusetts model},
author={Comeau, Audrey M. and Larson, Chad and Eaton, Robert B.},
journal={Pediatrics},
volume={114},
number={5},
pages={1370--1375},
year={2004},
publisher={American Academy of Pediatrics},
doi={10.1542/peds.2004-0710}
}`,
`@article{Southern2009,
title={Newborn screening for cystic fibrosis},
author={Southern, Kevin W. and Mérelle, Marieke M. E. and Dankert-Roelse, Jeannette E. and Nagelkerke, Nico},
journal={Cochrane Database of Systematic Reviews},
volume={1},
pages={CD001402},
year={2009},
publisher={John Wiley \& Sons},
doi={10.1002/14651858.CD001402.pub2}
}`,
`@article{Cutting2015,
title={Cystic fibrosis genetics: from molecular understanding to clinical application},
author={Cutting, Garry R.},
journal={Nature Reviews Genetics},
volume={16},
pages={45--56},
year={2015},
publisher={Nature Publishing Group},
doi={10.1038/nrg3849}
}`,
/* 61 */
`@article{Crossley1979,
title={Dried-blood spot screening for cystic fibrosis in the newborn},
author={Crossley, John R. and Elliott, Robert B. and Smith, Patricia A.},
journal={The Lancet},
volume={1},
number={8126},
pages={472--474},
year={1979},
publisher={Elsevier},
doi={10.1016/S0140-6736(79)91757-9}
}`,
`@book{Paracchini2011id,
title = "Cystic fibrosis newborn screening: Distribution of blood
immunoreactive trypsinogen concentrations in hypertrypsinemic
neonates",
booktitle = "{JIMD} Reports",
author = "Paracchini, Valentina and Seia, Manuela and Raimondi, Sara and
Costantino, Lucy and Capasso, Patrizia and Porcaro, Luigi and
Colombo, Carla and Coviello, Domenico A and Mariani, Tiziana and
Manzoni, Emanuela and Sangiovanni, Monica and Corbetta, Carlo",
publisher = "Springer Berlin Heidelberg",
pages = "17--23",
series = "JIMD reports",
year = 2011,
address = "Berlin, Heidelberg"
} `,
`@article{southern2007survey,
title={A survey of newborn screening for cystic fibrosis in Europe},
author={Southern, Kevin W and Munck, Anne and Pollitt, Rodney and Travert, Georges and Zanolla, Luisa and Dankert-Roelse, Jeannette and Castellani, Carlo and ECFS CF Neonatal Screening Working Group and others},
journal={Journal of Cystic Fibrosis},
volume={6},
number={1},
pages={57--65},
year={2007},
publisher={Elsevier}
} `,
/* 64)*/
`@ARTICLE{Mishra2008-ia,
title = "Diagnosis of cystic fibrosis by sweat testing: age-specific
reference intervals",
author = "Mishra, Avantika and Greaves, Ronda and Smith, Katherine and
Carlin, John B and Wootton, Andrew and Stirling, Rob and Massie,
John",
abstract = "OBJECTIVE: To develop reference intervals (RIs) for sweat
chloride and sodium in healthy children, adolescents, and
adults. STUDY DESIGN: Healthy, unrelated subjects aged from 5 to
>50 years and subjects who were pancreatic insufficient with
cystic fibrosis (CF) were recruited. Sweat collection was
performed on all subjects with the Wescor Macroduct system.
Sweat electrolytes were analyzed with direct ion selective
electrodes. DeltaF508 mutation analysis was performed on the
healthy subjects >/=15 years old. RESULTS: A total of 282
healthy and 40 subjects with CF were included for analysis.
There was no overlap of sweat chloride between the group with CF
and the group without CF, but there was some overlap of sweat
sodium. Sweat chloride increased with age, with the rate of
increase slowing progressively to zero after the age of 19
years. The estimated median (95\% RI) for sweat chloride were: 5
to 9 years, 13 mmol/L (1-39 mmol/L); 10 to 14 years, 18mmol/L
(3-47 mmol/L); 15 to 19 years, 20 mmol/L (3-51mmol/L); and 20+
years 23 mmol/L (5-56mmol/L). CONCLUSIONS: We have successfully
developed the age-related RI for sweat electrolytes, which will
be useful for clinicians interpreting sweat test results from
children, adolescents, and adults.",
journal = "J. Pediatr.",
publisher = "Elsevier BV",
volume = 153,
number = 6,
pages = "758--763",
month = dec,
year = 2008,
language = "en"
} `,
`
@misc{efwefwe,
author = {Cystic Fibrosis Foundation},
title = {Newborn Screening for CF},
url = {https://www.cff.org/intro-cf/newborn-screening-cf},
note = {Accessed on 02. October 2024}
`,`
@article{genes11040401,
title = {
The Impact of the CFTR Gene Discovery on Cystic Fibrosis Diagnosis,
Counseling, and Preventive Therapy
},
author = {Farrell, Philip M. and Rock, Michael J. and Baker, Mei W.},
year = 2020,
journal = {Genes},
volume = 11,
number = 4,
doi = {10.3390/genes11040401},
issn = {2073-4425},
url = {https://www.mdpi.com/2073-4425/11/4/401},
article-number = 401,
pubmedid = 32276344
}`,`
@article{farrell2020impact,
title = {The impact of the CFTR gene discovery on cystic fibrosis diagnosis, counseling, and preventive therapy},
author = {Farrell, Philip M and Rock, Michael J and Baker, Mei W},
year = 2020,
journal = {Genes},
publisher = {MDPI},
volume = 11,
number = 4,
pages = 401
}`,`
@article{REISMAN1988632,
title = {Role of conventional physiotherapy in cystic fibrosis},
author = {Reisman, John J. and Rivington-Law, Betty and Corey, Mary and Marcotte, Jacques and Wannamaker, Eleanor and Harcourt, Dawn and Levison, Henry},
year = 1988,
journal = {The Journal of Pediatrics},
volume = 113,
number = 4,
pages = {632--636},
doi = {https://doi.org/10.1016/S0022-3476(88)80370-6},
issn = {0022-3476},
url = {https://www.sciencedirect.com/science/article/pii/S0022347688803706}
}`,`
@article{Wainwright2015,
title = {Lumacaftor–ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR},
author = {Wainwright, Claire E and Elborn, J Stuart and Ramsey, Bonnie W and Marigowda, Girish and Huang, Xinxin and Cipolli, Marco and Colombo, Carlo and Davies, Jane C and De Boeck, Kris and Flume, Patrick A and others},
year = 2015,
journal = {New England Journal of Medicine},
publisher = {Massachusetts Medical Society},
volume = 373,
number = 3,
pages = {220--231},
doi = {10.1056/NEJMoa1409547}
}`,`
@article{Geller658,
title = {Aerosol Antibiotics in Cystic Fibrosis},
author = {Geller, David E},
year = 2009,
journal = {Respiratory Care},
publisher = {Respiratory Care},
volume = 54,
number = 5,
pages = {658--670},
url = {https://rc.rcjournal.com/content/54/5/658},
ssn = {0020-1324},
eprint = {https://rc.rcjournal.com/content/54/5/658.full.pdf}
}`,`
@article{Tummler2016,
title = {Therapie der Mukoviszidose mit CFTR-Modulatoren},
author = {Tümmler, B.},
year = 2016,
journal = {Pneumologie},
publisher = {Georg Thieme Verlag KG},
volume = 70,
number = 3,
pages = {301--313},
doi = {10.1055/s-0042-100607},
issn = {0934-8387},
url = {https://www.thieme-connect.com/products/ejournals/pdf/10.1055/s-0042-100607.pdf}
}`,`
@misc{MukoTrikafta2024internet,
title = {Kaftrio (Trikafta) – Triple-Therapie für Mukoviszidose (CF)},
author = {Mukoviszidose e.V.},
year = 2024,
url = {https://www.muko.info/mukoviszidose/therapie-der-mukoviszidose/kaftrio-trikafta​},
note = {Zugriff am 07. August 2024}
}
`,
/* 66 */
`@book{Hegyi2016,
author={Hegyi, Péter and Wilschanski, Michael and Muallem, Shmuel and Lukacs, Gergely L. and Sahin-Tóth, Miklós and Uc, Aliye and Gray, Michael A. and Rakonczay, Zoltán and Maléth, József},
editor={Nilius, Bernd and de Tombe, Pieter and Gudermann, Thomas and Jahn, Reinhard and Lill, Roland and Petersen, Ole H.},
title={CFTR: A New Horizon in the Pathomechanism and Treatment of Pancreatitis},
bookTitle={Reviews of Physiology, Biochemistry and Pharmacology Vol. 170},
year={2016},
publisher={Springer International Publishing},
address={Cham},
pages={37--66},
isbn={978-3-319-31492-1},
doi={10.1007/112_2015_5002},
url={https://doi.org/10.1007/112_2015_5002}
}`,
`@article{farrell2020impact,
title={The impact of the CFTR gene discovery on cystic fibrosis diagnosis, counseling, and preventive therapy},
author={Farrell, Philip M and Rock, Michael J and Baker, Mei W},
journal={Genes},
volume={11},
number={4},
pages={401},
year={2020},
publisher={MDPI}
}`,
`@article{Flume2009,
title={Cystic fibrosis pulmonary guidelines: airway clearance therapies},
author={Flume, Patrick A. and Robinson, Karen A. and O'Sullivan, Brian P and Finder, Jon D and Vender, Robin L and Willey-Courand, Donna B and White, Thomas B and Marshall, Bruce C.},
journal={Respiratory Care},
volume={54},
number={4},
pages={522--537},
year={2009},
publisher={Respiratory Care},
doi={10.4187/aarc0555}
}`,
`@article{REISMAN1988632,
title={Role of conventional physiotherapy in cystic fibrosis},
journal={The Journal of Pediatrics},
volume={113},
number={4},
pages={632-636},
year={1988},
issn={0022-3476},
doi={https://doi.org/10.1016/S0022-3476(88)80370-6},
url={https://www.sciencedirect.com/science/article/pii/S0022347688803706},
author={Reisman, John J. and Rivington-Law, Betty and Corey, Mary and Marcotte, Jacques and Wannamaker, Eleanor and Harcourt, Dawn and Levison, Henry}
}`,
`@article{Wainwright2015,
title={Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR},
author={Wainwright, Claire E and Elborn, J Stuart and Ramsey, Bonnie W and Marigowda, Girish and Huang, Xinxin and Cipolli, Marco and Colombo, Carlo and Davies, Jane C and De Boeck, Kris and Flume, Patrick A and others},
journal={New England Journal of Medicine},
volume={373},
number={3},
pages={220--231},
year={2015},
publisher={Massachusetts Medical Society},
doi={10.1056/NEJMoa1409547}
}`,
`@article{Geller658,
author={Geller, David E},
title={Aerosol Antibiotics in Cystic Fibrosis},
volume={54},
number={5},
pages={658--670},
year={2009},
publisher={Respiratory Care},
issn={0020-1324},
url={https://rc.rcjournal.com/content/54/5/658},
eprint={https://rc.rcjournal.com/content/54/5/658.full.pdf},
journal={Respiratory Care}
}`,
`@ARTICLE{Guo2022-ch,
title={Worldwide rates of diagnosis and effective treatment for cystic fibrosis},
author={Guo, Jonathan and Garratt, Anna and Hill, Andrew},
journal={Journal of Cystic Fibrosis},
volume={21},
number={3},
pages={456--462},
year={2022},
month={may},
doi={10.1016/j.jcf.2021.12.001},
keywords={CFTR modulator, Cystic fibrosis, Epidemiology, Ivacaftor/tezacaftor/elexacaftor},
publisher={Elsevier BV}
}`,
`@article{Griesenbach2013,
title={Gene therapy progress and prospects: cystic fibrosis},
author={Griesenbach, Uta and Alton, Eric WFW},
journal={Gene Therapy},
volume={20},
number={6},
pages={641--649},
year={2013},
publisher={Nature Publishing Group},
doi={10.1038/gt.2013.20}
}`,
`@misc{Muko52024internet,
author={Mukoviszidose e.V},
title={Therapie der Mukoviszidose},
year={2024},
url={https://www.muko.info/mukoviszidose/therapie-der-mukoviszidose},
note={Zugriff am 07. August 2024}
}`,
/* 75 */
`@article{jia2023cystic,
author={Jia, Shijing and Taylor-Cousar, Jennifer L.},
title={Cystic Fibrosis Modulator Therapies},
journal={Annual Review of Medicine},
volume={74},
pages={413-426},
year={2023},
doi={10.1146/annurev-med-042921-021447},
url={https://www.annualreviews.org/content/journals/10.1146/annurev-med-042921-021447},
publisher={Annual Reviews},
issn={1545-326X}
}`,
`@article{Tummler2016,
author={Tümmler, B.},
title={Therapie der Mukoviszidose mit CFTR-Modulatoren},
journal={Pneumologie},
volume={70},
number={3},
pages={301-313},
year={2016},
doi={10.1055/s-0042-100607},
url={https://www.thieme-connect.com/products/ejournals/pdf/10.1055/s-0042-100607.pdf},
publisher={Georg Thieme Verlag KG},
issn={0934-8387}
}`,
`@misc{Muko42024internet,
author={Mukoviszidose e.V},
title={Kaftrio (Trikafta) - Triple-Therapie für Mukoviszidose (CF)},
year={2024},
url={https://www.muko.info/mukoviszidose/therapie-der-mukoviszidose/kaftrio-trikafta},
note={Zugriff am 07. August 2024}
}`,
`@article{roda2022new,
author={Roda, Juliana and Pinto-Silva, Catarina and Silva, Iris A. I. and Maia, Carla and Almeida, Susana and Ferreira, Ricardo and Oliveira, Guiomar},
title={New drugs in cystic fibrosis: what has changed in the last decade?},
journal={Therapeutic Advances in Chronic Disease},
volume={13},
pages={20406223221098136},
year={2022},
doi={10.1177/20406223221098136},
pmid={35620188},
pmc={PMC9128052},
issn={2040-6223},
note={eCollection 2022},
publisher={SAGE Publications},
url={https://doi.org/10.1177/20406223221098136}
}`,
`@misc{Muko32024internet,
author={Mukoviszidose e.V},
title={Orkambi - CFTR-Modulator bei zwei F508del-Mutationen},
year={2024},
url={https://www.muko.info/mukoviszidose/therapie-der-mukoviszidose/orkambi},
note={Zugriff am 07. August 2024}
}`,
`@misc{Muko22024internet,
author={Mukoviszidose e.V},
title={Kalydeco (Ivafactor) - CFTR-Modulator bei Gating-Mutationen},
year={2024},
url={https://www.muko.info/mukoviszidose/therapie-der-mukoviszidose/kalydeco},
note={Zugriff am 07. August 2024}
}`,
/* 81 */
`@misc{Pulmo2024internet,
author={Pulmonyze dornase alfa Inhalation Solution},
title={What is Pulmonyze?},
year={2019},
url={https://www.pulmozyme.global/home.html},
note={Zugriff am 07. August 2024}
}`,
`@misc{pulmozyme_approval_CDER,
author={Center for Drug Evaluation and Research (CDER)},
title={CDER List of Licensed Biological Products with Reference Product Exclusivity and Biosimilarity or Interchangeability Evaluations},
year={2019},
url={file:///mnt/data/CDER%20List%20of%20Licensed%20Biological%20Products%204-24-2019_.pdf}
}`,
`@misc{CFF22024internet,
author={Cystic Fibrosis Foundation},
title={Mucus Thinners},
year={2024},
url={https://www.cff.org/managing-cf/mucus-thinners},
note={Zugriff am 07. August 2024}
}`,
/* 84 */
`@article{Terlizzi2021,
title={Hypertonic saline in people with cystic fibrosis: review of comparative studies and clinical practice},
author={Terlizzi, Valeria and Masi, Eleonora and Francalanci, Marco and Braggion, Chiara},
journal={Italian Journal of Pediatrics},
volume={47},
number={1},
pages={168},
year={2021},
publisher={Springer},
doi={10.1186/s13052-021-01117-1},
url={https://ijponline.biomedcentral.com/articles/10.1186/s13052-021-01117-1}
}`,
`@misc{CFF12024internet,
author={Cystic Fibrosis Foundation},
title={Antibiotics},
year={2024},
url={https://www.cff.org/managing-cf/antibiotics},
note={Zugriff am 07. August 2024}
}`,
`@misc{TOBI2024internet,
author={TOBI® Podhaler®},
title={TOBI® Podhaler®},
year={2024},
url={https://www.tobipodhaler.com/},
note={Zugriff am 07. August 2024}
}`,
`@misc{EMA22024internet,
author={European Medicines Agency},
title={Tobi Podhaler},
year={2024},
url={https://www.ema.europa.eu/en/medicines/human/EPAR/tobi-podhaler},
note={Zugriff am 07. August 2024}
}`,
`@misc{EMA12024internet,
author={European Medicines Agency},
title={Cayston},
year={2024},
url={https://www.ema.europa.eu/en/medicines/human/EPAR/cayston},
note={Zugriff am 07. August 2024}
}`,
/* 89 */
`@misc{Muko12024internet,
author={Mukoviszidose e.V},
title={Therapie der Bauchspeicheldrüsenfehlfunktion (Pankreasinsuffizienz): Enzymsubstitution},
year={2024},
url={https://www.muko.info/mitwirken/arbeitskreise-und-arbeitsgemeinschaften/ak-ernaehrung/ernaehrungstherapie-bei-mukoviszidose/pankreasinsuffizienz-und-enzymsubstitution},
note={Zugriff am 07. August 2024}
}`,
/* 90 */
`@misc{CREON2024internet,
author={CREON® (pancrelipase)},
title={About CREON®},
year={2024},
url={https://www.creonhcp.com/about-creon},
note={Zugriff am 07. August 2024}
}`,
/* original 91 ist 15 */
`@misc{BroadInstitute2024,
author = {Broad Institute of MIT and Harvard},
title = {Researchers engineer in vivo delivery system for prime editing, partially restoring vision in mice},
publisher = {Phys.org},
year = {2024},
`,`
@misc{muko2024therapie,
title = {Therapie der Mukoviszidose},
author = {Mukoviszidose e.V.},
year = 2024,
url = {https://www.muko.info/mukoviszidose/therapie-der-mukoviszidose},
note = {Zugriff am 7. August 2024}
}`,`
@article{Jia2023,
title = {Cystic Fibrosis Modulator Therapies},
author = {Jia, Shijing and Taylor-Cousar, Jennifer L.},
year = 2023,
journal = {Annual Review of Medicine},
volume = 74,
pages = {413--426},
doi = {10.1146/annurev-med-042921-021447},
url = {https://doi.org/10.1146/annurev-med-042921-021447}
}`,`
@article{Tuemmler2016,
title = {Therapie der Mukoviszidose mit CFTR-Modulatoren},
author = {Tümmler, Burkhard},
year = 2016,
journal = {Pneumologie},
volume = 70,
number = 5,
pages = {301--313},
doi = {10.1055/s-0042-100607},
url = {
https://www.thieme-connect.com/products/ejournals/html/10.1055/s-0042-100607
}
}`,`
@article{doihbjhb,
title = {New drugs in cystic fibrosis: what has changed in the last decade?},
author = {
Roda, Juliana and Pinto-Silva, Catarina and Silva, Iris A.I. and Maia, Carla and
Almeida, Susana and Ferreira, Ricardo and Oliveira, Guiomar
},
year = 2022,
journal = {Therapeutic Advances in Chronic Disease},
volume = 13,
pages = 20406223221098136,
doi = {10.1177/20406223221098136},
url = {https://doi.org/10.1177/20406223221098136},
note = {PMID: 35620188},
eprint = {https://doi.org/10.1177/20406223221098136}
}`,`
@misc{cff2024antibiotics,
title = {Managing CF: Antibiotics},
author = {Cystic Fibrosis Foundation},
year = 2024,
url = {https://www.cff.org/managing-cf/antibiotics},
note = {Zugriff am 7. August 2024}
}`,`
@misc{ema2024tobi,
title = {Tobi Podhaler: EPAR - Summary for the Public},
author = {European Medicines Agency (EMA)},
year = 2024,
url = {https://www.ema.europa.eu/en/medicines/human/EPAR/tobi-podhaler},
note = {Zugriff am 7. August 2024}
}`,`
@misc{muko2024pankreas,
title = {Pankreasinsuffizienz und Enzymsubstitution},
author = {Mukoviszidose e.V.},
year = 2024,
url = {
https://www.muko.info/mitwirken/arbeitskreise-und-arbeitsgemeinschaften/ak-ernaehrung/ernaehrungstherapie-bei-mukoviszidose/pankreasinsuffizienz-und-enzymsubstitution
},
note = {Zugriff am 7. August 2024}
}`,`
@article{hjbhjgj,
title = {Search-and-replace genome editing without double-strand breaks or donor DNA},
author = {Anzalone, Andrew V. and Randolph, Peyton B. and Davis, Jessie R. and Sousa, Alexander A. and Koblan, Luke W. and Levy, Jonathan M. and Chen, Peter J. and Wilson, Christopher and Newby, Gregory A. and Raguram, Aditya and Liu, David R.},
year = 2019,
journal = {Nature},
volume = 576,
number = 7785,
pages = {149--157},
doi = {10.1038/s41586-019-1711-4}
}
`,
`@article{Badon_Oh_Kim_Lee_2024, title={Recent application of CRISPR-Cas12 and OMEGA system for genome editing}, volume={32}, ISSN={1525-0016, 1525-0024}, DOI={10.1016/j.ymthe.2023.11.013}, number={1}, journal={Molecular Therapy}, publisher={Elsevier}, author={Badon, Isabel Wen and Oh, Yeounsun and Kim, Ho-Joong and Lee, Seung Hwan}, year={2024}, month=jan, pages={32–43}, language={English} } `,
/* 94 */
`@article{Saito_Xu_Faure_Maguire_Kannan_Altae-Tran_Vo_Desimone_Macrae_Zhang_2023,
title={Fanzor is a eukaryotic programmable RNA-guided endonuclease},
volume={620},
rights={2023 The Author(s)},
ISSN={1476-4687},
DOI={10.1038/s41586-023-06356-2},
number={7974},
journal={Nature},
publisher={Nature Publishing Group},
author={Saito, Makoto and Xu, Peiyu and Faure, Guilhem and Maguire, Samantha and Kannan, Soumya and Altae-Tran, Han and Vo, Sam and Desimone, AnAn and Macrae, Rhiannon K. and Zhang, Feng},
year={2023},
month={aug},
pages={660-668},
language={en}
}`,
`@article{Liu_Orlova_Oakes_Ma_Spinner_Baney_Chuck_Tan_Knott_,
title={CasX enzymes comprise a distinct family of RNA-guided genome editors},
volume={566},
rights={2019 The Author(s), under exclusive licence to Springer Nature Limited},
ISSN={1476-4687},
DOI={10.1038/s41586-019-0908-x},
number={7743},
journal={Nature},
publisher={Nature Publishing Group},
author={Liu, Jun-Jie and Orlova, Natalia and Oakes, Benjamin L. and Ma, Enbo and Spinner, Hannah B. and Baney, Katherine L. M. and Chuck, Jonathan and Tan, Dan and Knott, Gavin J. and Harrington, Lucas B. and Al-Shayeb, Basem and Wagner, Alexander and Brötzmann, Julian and Staahl, Brett T. and Taylor, Kian L. and Desmarais, John and Nogales, Eva and Doudna, Jennifer A.},
year={2019},
month={feb},
pages={218-223},
language={en}
}`,
`@article{Doman_Pandey_Neugebauer_An_Davis_Randolph_McElroy_Gao_Ragura,
title = {Phage-assisted evolution and protein engineering yield compact, efficient prime editors},
author = {Doman, J. L. and Pandey, S. and Neugebauer, M. E. and An, Meirui and Davis, Jessie R. and Randolph, Peyton B. and McElroy, Amber and Gao, Xin D. and Raguram, Aditya and Richter, Michelle F. and Everette, Kelcee A. and Banskota, Samagya and Tian, Kathryn and Tao, Y. Allen and Tolar, Jakub and Osborn, Mark J. and Liu, David R.},
`,`
@article{ivpeoamldim2022,
title = {In vivo prime editing of a metabolic liver disease in mice},
author = {
Böck D, Rothgangl T, Villiger L, Schmidheini L, Matsushita M, Mathis N,
Ioannidi E, Rimann N, Grisch-Chan HM, Kreutzer S, Kontarakis Z, Kopf M, Thöny
B, Schwank G
},
year = 2022,
journal = {PupMed},
volume = 14,
number = 1,
pages = {eabl9238},
doi = {10.1126/scitranslmed.abl9238},
url = {https://pmc.ncbi.nlm.nih.gov/articles/PMC7614134/}
}`,`
@article{CasX_enzymes_comprise_a_distinct_family_of_RNA-guided_genome_editors,
title = {CasX enzymes comprise a distinct family of RNA-guided genome editors},
author = {Liu, Jun-Jie and Orlova, Natalia and Oakes, Benjamin L. and Ma, Enbo and Spinner, Hannah B. and Baney, Katherine L. M. and Chuck, Jonathan and Tan, Dan and Knott, Gavin J. and Harrington, Lucas B. and Al-Shayeb, Basem and Wagner, Alexander and Brötzmann, Julian and Staahl, Brett T. and Taylor, Kian L. and Desmarais, John and Nogales, Eva and Doudna, Jennifer A.},
year = 2019,
journal = {Nature},
volume = 566,
number = 7743,
pages = 218,
doi = {10.1038/s41586-019-0908-x}
}`,`
@article{Fanzor_is_a_eukaryotic_programmable_RNA-guided_endonuclease,
title = {Fanzor is a eukaryotic programmable RNA-guided endonuclease},
author = {Saito, Makoto and Xu, Peiyu and Faure, Guilhem and Maguire, Samantha and Kannan, Soumya and Altae-Tran, Han and Vo, Sam and Desimone, AnAn and Macrae, Rhiannon K. and Zhang, Feng},
year = 2023,
journal = {Cell},
publisher = {Elsevier},
volume = 186,
number = 18,
pages = {3983--4002},
doi = {10.1016/j.cell.2023.07.039},
language = {English}
journal = {Nature},
volume = 620,
number = 7974,
pages = 660,
doi = {10.1038/s41586-023-06356-2}
}
`,
`@article{IWAWAKI2006709,
title={Analysis of the XBP1 splicing mechanism using endoplasmic reticulum stress-indicators},
journal={Biochemical and Biophysical Research Communications},
volume={350},
number={3},
pages={709-715},
year={2006},
ISSN={0006-291X},
DOI={https://doi.org/10.1016/j.bbrc.2006.09.100},
url={https://www.sciencedirect.com/science/article/pii/S0006291X06021553},
author={Iwawaki, Takao and Akai, Ryoko},
keywords={ER stress, IRE1, Splicing, Unfolded protein response, XBP1}
}`,
]
\ No newline at end of file
`,`
@article{Ma2024,
title = {A Perspective of Lipid Nanoparticles for RNA Delivery},
author = {Ma, Yutian and Li, Shiyao and Lin, Xin and Chen, Yupeng},
year = 2024,
journal = {Exploration},
volume = 4,
number = 1,
pages = 20230147,
doi = {10.1002/EXP.20230147},
url = {https://onlinelibrary.wiley.com/doi/10.1002/EXP.20230147}
}`,`
@article{Sahay2021,
title = {Lipid nanoparticles for mRNA delivery},
author = {Sahay, Gaurav and Querbes, Jordan and Gupta, Amit K. and Stanton-Maxey, Benjamin Z. and Davis, Mark E. and Szoka, Frank C. and Anderson, Daniel G.},
year = 2021,
journal = {Nature Reviews Materials},
volume = 6,
pages = {1078--1094},
doi = {10.1038/s41578-021-00358-0},
url = {https://www.nature.com/articles/s41578-021-00358-0}
}
`,`
@article{NEARY2024812,
title = {
Nebulised delivery of RNA formulations to the lungs: From aerosol to cytosol
},
author = {Neary, Michael T. and Mulder, Lianne M. and Kowalski, Piotr S. and MacLoughlin, Ronan and Crean, Abina M. and Ryan, Katie B.},
year = 2024,
journal = {Journal of Controlled Release},
volume = 366,
pages = {812--833},
doi = {https://doi.org/10.1016/j.jconrel.2023.12.012},
issn = {0168-3659},
url = {https://www.sciencedirect.com/science/article/pii/S0168365923007964}
}`,`
@article{WANG2024105547,
title = {
The optimization strategies of LNP-mRNA formulations: Development and
challenges for further application
},
author = {Wang, Sizhen and Guo, Beibei and Wang, Huan and Yang, Feng},
year = 2024,
journal = {Journal of Drug Delivery Science and Technology},
volume = 95,
pages = 105547,
doi = {https://doi.org/10.1016/j.jddst.2024.105547},
issn = {1773-2247},
url = {https://www.sciencedirect.com/science/article/pii/S1773224724002156}
}`,`
@article{TAHA2022345,
title = {
Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and
challenges
},
author = {Taha, Eman A. and Lee, Joseph and Hotta, Akitsu},
year = 2022,
journal = {Journal of Controlled Release},
volume = 342,
pages = {345--361},
doi = {https://doi.org/10.1016/j.jconrel.2022.01.013},
issn = {0168-3659},
url = {https://www.sciencedirect.com/science/article/pii/S016836592200027X}
}`,`
@article{LOPRESTI2022819,
title = {
The replacement of helper lipids with charged alternatives in lipid
nanoparticles facilitates targeted mRNA delivery to the spleen and lungs
},
author = {
LoPresti, Samuel T. and Arral, Mariah L. and Chaudhary, Namit and Whitehead, Kathryn A.
},
year = 2022,
journal = {Journal of Controlled Release},
volume = 345,
pages = {819--831},
doi = {https://doi.org/10.1016/j.jconrel.2022.03.046},
issn = {0168-3659},
url = {https://www.sciencedirect.com/science/article/pii/S0168365922001808}
}`,`
@article{pharmaceutics15030772,
title = {
Recent Progress of Lipid Nanoparticles-Based Lipophilic Drug Delivery: Focus
on Surface Modifications
},
author = {
Seo, Yoseph and Lim, Hayeon and Park, Hyunjun and Yu, Jiyun and An, Jeongyun
and Yoo, Hah Young and Lee, Taek
},
year = 2023,
journal = {Pharmaceutics},
volume = 15,
number = 3,
doi = {10.3390/pharmaceutics15030772},
issn = {1999-4923},
url = {https://www.mdpi.com/1999-4923/15/3/772},
article-number = 772,
pubmedid = 36986633
}`,`
@article{YAMAMOTO2005373,
title = {
Surface-modified PLGA nanosphere with chitosan improved pulmonary delivery of
calcitonin by mucoadhesion and opening of the intercellular tight junctions
},
author = {
Yamamoto, Hiromitsu and Kuno, Yoshio and Sugimoto, Shohei and Takeuchi, Hirofumi and Kawashima, Yoshiaki
},
year = 2005,
journal = {Journal of Controlled Release},
volume = 102,
number = 2,
pages = {373--381},
doi = {https://doi.org/10.1016/j.jconrel.2004.10.010},
issn = {0168-3659},
url = {https://www.sciencedirect.com/science/article/pii/S0168365904004912}
}`,`
@article{Hashiba2024,
title = {
Overcoming thermostability challenges in mRNA-lipid nanoparticle systems with
piperidine-based ionizable lipids
},
author = {
Hashiba, Kazuki and Taguchi, Masamitsu and Sakamoto, Sachiko and Otsu, Ayaka and Maeda, Yoshiki and Ebe, Hirofumi and Okazaki, Arimichi and Harashima, Hideyoshi and Sato, Yusuke
},
year = 2024,
journal = {Communications Biology},
volume = 7,
pages = 556,
doi = {10.1038/s42003-024-06235-0},
url = {https://www.nature.com/articles/s42003-024-06235-0}
}`,`
@article{Schober2024,
title = {
A careful look at lipid nanoparticle characterization: analysis of benchmark
formulations for encapsulation of RNA cargo size gradient
},
author = {Schober, Gretchen B. and Story, Sandra and Arya, Dev P.},
year = 2024,
journal = {Scientific Reports},
volume = 14,
pages = 2403,
doi = {10.1038/s41598-024-52685-1},
url = {https://www.nature.com/articles/s41598-024-52685-1}
}`,`
@article{Cullis2017,
title = {Lipid Nanoparticle Systems for Enabling Gene Therapies},
author = {Cullis, Pieter R. },
year = 2017,
journal = {Molecular Therapy},
volume = 25,
number = 7,
pages = {1463--1466},
doi = {10.1016/j.ymthe.2017.03.027},
url = {
https://www.cell.com/molecular-therapy-family/molecular-therapy/fulltext/S1525-0016(17)30111-9
}
}`,`
@article{Kim2022,
title = {
Engineering Lipid Nanoparticles for Enhanced Intracellular Delivery of mRNA
through Inhalation
},
author = {
Kim, Jeonghwan and Jozic, Antony and Lin, Yuxin and Eygeris, Yulia and Bloom, Elissa and Tan, Xiaochen and Acosta, Christopher and MacDonald, Kelvin D. and Welsher, Kevin D. and Sahay, Gaurav
},
year = 2022,
journal = {Proceedings of the National Academy of Sciences},
volume = 119,
number = 36,
pages = {e2202539119},
doi = {10.1073/pnas.2202539119},
url = {https://www.pnas.org/doi/10.1073/pnas.2202539119}
}`,`
@article{Wang2023,
title = {
Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs)
using multiple technical methods for tissue-specific mRNA delivery
},
author = {
Wang, Xu and Liu, Shuai and Sun, Yehui and Yu, Xueliang and Lee, Sang M. and Cheng, Qiang and Wei, Tuo and Gong, Junyu and Robinson, Joshua and Zhang, Di and Lian, Xizhen and Basak, Pratima and Siegwart, Daniel J.
},
year = 2023,
journal = {Nature Protocols},
volume = 18,
pages = {265--291},
doi = {10.1038/s41596-022-00755-x},
url = {https://doi.org/10.1038/s41596-022-00755-x}
}`,`
@article{Wei2023,
title = {
Lung SORT LNPs enable precise homology-directed repair mediated CRISPR/Cas
genome correction in cystic fibrosis models
},
author = {
Wei, Tuo and Sun, Yehui and Cheng, Qiang and Chatterjee, Sumanta and Traylor, Zachary and Johnson, Lindsay T. and Coquelin, Melissa L. and Wang, Jialu and Torres, Michael J. and Lian, Xizhen and Wang, Xu and Xiao, Yufen and Hodges, Craig A. and Siegwart, Daniel J.
},
year = 2023,
journal = {Nature Communications},
volume = 14,
pages = 7322,
doi = {10.1038/s41467-023-42948-2},
url = {https://doi.org/10.1038/s41467-023-42948-2}
}`,`
@article{ZIMMERMANN2022137,
title = {Spray drying siRNA-lipid nanoparticles for dry powder pulmonary delivery},
author = {
Zimmermann, Christoph M. and Baldassi, Domizia and Chan, Karen and Adams, Nathan B.P. and Neumann, Alina and Porras-Gonzalez, Diana Leidy and Wei, Xin and Kneidinger, Nikolaus and Stoleriu, Mircea Gabriel and Burgstaller, Gerald and Witzigmann, Dominik and Luciani, Paola and Merkel, Olivia M.
},
year = 2022,
journal = {Journal of Controlled Release},
volume = 351,
pages = {137--150},
doi = {https://doi.org/10.1016/j.jconrel.2022.09.021},
issn = {0168-3659},
url = {https://www.sciencedirect.com/science/article/pii/S0168365922006137},
keywords = {
Lipid nanoparticles, LNP, Onpattro®, RNA therapeutics, siRNA delivery, Spray
drying, Pulmonary delivery, Respiratory diseases, Human precision-cut lung
slices, Formulation screening
}
}`,`
@article{IWAWAKI2006709,
title = {
Analysis of the XBP1 splicing mechanism using endoplasmic reticulum
stress-indicators
},
author = {Iwawaki, Takao and Akai, Ryoko},
year = 2006,
journal = {Biochemical and Biophysical Research Communications},
volume = 350,
number = 3,
pages = {709--715},
doi = {https://doi.org/10.1016/j.bbrc.2006.09.100},
issn = {0006-291X},
url = {https://www.sciencedirect.com/science/article/pii/S0006291X06021553},
keywords = {ER stress, IRE1, Splicing, Unfolded protein response, XBP1}
}
`
]
src/sources/eng-delivery-sources.tsx
View file @ 3fd292fd
......@@ -3,7 +3,7 @@ import BibtexParser from "../components/makeSources";
export default function EngDelsources(){
return (
<div>
<BibtexParser bibtexSources={bibtexSources} />
<BibtexParser bibtexSources={bibtexSources} start={30}/>
</div>
);
}
......@@ -77,7 +77,7 @@ const bibtexSources = [
}
`,`
@article{wei_lung_2023,
title = {Lung {SORT} {LNPs} enable precise homology-directed repair mediated {CRISPR}/{Cas} genome correction in cystic fibrosis models},
title = {Lung {SORT} {LNPs} enable precise homology-directed repair mediated {CRISPR}/{Cas} genome correction in Cystic Fibrosis models},
author = {Wei, Tuo and Sun, Yehui and Cheng, Qiang and Chatterjee, Sumanta and Traylor, Zachary and Johnson, Lindsay T. and Coquelin, Melissa L. and Wang, Jialu and Torres, Michael J. and Lian, Xizhen and Wang, Xu and Xiao, Yufen and Hodges, Craig A. and Siegwart, Daniel J.},
year = 2023,
month = nov,
......@@ -121,7 +121,7 @@ const bibtexSources = [
issn = {0168-3659},
url = {https://www.sciencedirect.com/science/article/pii/S0168365922006137},
keywords = {Lipid nanoparticles, LNP, Onpattro®, RNA therapeutics, siRNA delivery, Spray drying, Pulmonary delivery, Respiratory diseases, Human precision-cut lung slices, Formulation screening},
abstract = {While all the siRNA drugs on the market target the liver, the lungs offer a variety of currently undruggable targets which could potentially be treated with RNA therapeutics. Hence, local, pulmonary delivery of RNA nanoparticles could finally enable delivery beyond the liver. The administration of RNA drugs via dry powder inhalers offers many advantages related to physical, chemical and microbial stability of RNA and nanosuspensions. The present study was therefore designed to test the feasibility of engineering spray dried lipid nanoparticle (LNP) powders. Spray drying was performed using 5% lactose solution (m/V), and the targets were set to obtain nanoparticle sizes after redispersion of spray-dried powders around 150 nm, a residual moisture level below 5%, and RNA loss below 15% at maintained RNA bioactivity. The LNPs consisted of an ionizable cationic lipid which is a sulfur-containing analog of DLin-MC3-DMA, a helper lipid, cholesterol, and PEG-DMG encapsulating siRNA. Prior to the spray drying, the latter process was simulated with a novel dual emission fluorescence spectroscopy method to preselect the highest possible drying temperature and excipient solution maintaining LNP integrity and stability. Through characterization of physicochemical and aerodynamic properties of the spray dried powders, administration criteria for delivery to the lower respiratory tract were fulfilled. Spray dried LNPs penetrated the lung mucus layer and maintained bioactivity for >90% protein downregulation with a confirmed safety profile in a lung adenocarcinoma cell line. Additionally, the spray dried LNPs successfully achieved up to 50% gene silencing of the house keeping gene GAPDH in ex vivo human precision-cut lung slices at without increasing cytokine levels. This study verifies the successful spray drying procedure of LNP-siRNA systems maintaining their integrity and mediating strong gene silencing efficiency on mRNA and protein levels both in vitro and ex vivo. The successful spray drying procedure of LNP-siRNA formulations in 5% lactose solution creates a novel siRNA-based therapy option to target respiratory diseases such as lung cancer, asthma, COPD, cystic fibrosis and viral infections.}
abstract = {While all the siRNA drugs on the market target the liver, the lungs offer a variety of currently undruggable targets which could potentially be treated with RNA therapeutics. Hence, local, pulmonary delivery of RNA nanoparticles could finally enable delivery beyond the liver. The administration of RNA drugs via dry powder inhalers offers many advantages related to physical, chemical and microbial stability of RNA and nanosuspensions. The present study was therefore designed to test the feasibility of engineering spray dried lipid nanoparticle (LNP) powders. Spray drying was performed using 5% lactose solution (m/V), and the targets were set to obtain nanoparticle sizes after redispersion of spray-dried powders around 150 nm, a residual moisture level below 5%, and RNA loss below 15% at maintained RNA bioactivity. The LNPs consisted of an ionizable cationic lipid which is a sulfur-containing analog of DLin-MC3-DMA, a helper lipid, cholesterol, and PEG-DMG encapsulating siRNA. Prior to the spray drying, the latter process was simulated with a novel dual emission fluorescence spectroscopy method to preselect the highest possible drying temperature and excipient solution maintaining LNP integrity and stability. Through characterization of physicochemical and aerodynamic properties of the spray dried powders, administration criteria for delivery to the lower respiratory tract were fulfilled. Spray dried LNPs penetrated the lung mucus layer and maintained bioactivity for >90% protein downregulation with a confirmed safety profile in a lung adenocarcinoma cell line. Additionally, the spray dried LNPs successfully achieved up to 50% gene silencing of the house keeping gene GAPDH in ex vivo human precision-cut lung slices at without increasing cytokine levels. This study verifies the successful spray drying procedure of LNP-siRNA systems maintaining their integrity and mediating strong gene silencing efficiency on mRNA and protein levels both in vitro and ex vivo. The successful spray drying procedure of LNP-siRNA formulations in 5% lactose solution creates a novel siRNA-based therapy option to target respiratory diseases such as lung cancer, asthma, COPD, Cystic Fibrosis and viral infections.}
}
`,`
@article{jiang_combinatorial_2024,
......@@ -154,7 +154,7 @@ const bibtexSources = [
url = {https://respiratory-research.biomedcentral.com/articles/10.1186/s12931-024-02800-7},
urldate = {2024-09-10},
abstract = {
Background Pulmonary ionocytes have been identified in the airway epithelium as a small population of ion transporting cells expressing high levels of CFTR (cystic fibrosis transmembrane conductance regulator), the gene mutated in cystic fibrosis. By providing an infinite source of airway epithelial cells (AECs), the use of human induced pluripotent stem cells (hiPSCs) could overcome some challenges of studying ionocytes. However, the production of AEC epithelia containing ionocytes from hiPSCs has proven difficult. Here, we present a platform to produce hiPSCderived AECs (hiPSC-AECs) including ionocytes and investigate their role in the airway epithelium.
Background Pulmonary ionocytes have been identified in the airway epithelium as a small population of ion transporting cells expressing high levels of CFTR ( Cystic Fibrosis transmembrane conductance regulator), the gene mutated in Cystic Fibrosis. By providing an infinite source of airway epithelial cells (AECs), the use of human induced pluripotent stem cells (hiPSCs) could overcome some challenges of studying ionocytes. However, the production of AEC epithelia containing ionocytes from hiPSCs has proven difficult. Here, we present a platform to produce hiPSCderived AECs (hiPSC-AECs) including ionocytes and investigate their role in the airway epithelium.
Methods hiPSCs were differentiated into lung progenitors, which were expanded as 3D organoids and matured by air-liquid interface culture as polarised hiPSC-AEC epithelia. Using CRISPR/Cas9 technology, we generated a hiPSCs knockout (KO) for FOXI1, a transcription factor that is essential for ionocyte specification. Differences between FOXI1 KO hiPSC-AECs and their wild-type (WT) isogenic controls were investigated by assessing gene and protein expression, epithelial composition, cilia coverage and motility, pH and transepithelial barrier properties.
......
src/sources/eng-nickases-sources.tsx
View file @ 3fd292fd
......@@ -3,7 +3,7 @@ import BibtexParser from "../components/makeSources";
export default function EngNicksources(){
return (
<div>
<BibtexParser bibtexSources={bibtexSources} />
<BibtexParser bibtexSources={bibtexSources} start={24}/>
</div>
);
}
......
src/sources/eng-pe-sources.tsx
View file @ 3fd292fd
......@@ -3,13 +3,13 @@ import BibtexParser from "../components/makeSources";
export default function EngPEsystems(){
return (
<div>
<BibtexParser bibtexSources={bibtexSources} />
<BibtexParser bibtexSources={bibtexSources} start={6}/>
</div>
);
}
const bibtexSources = [
const bibtexSources = [ /* 1-> 6 */
`@article{Anzalone_Randolph_Davis_Sousa_Koblan_Levy_Chen_Wilson_Newby_Raguram_2019,
title = {Search-and-replace genome editing without double-strand breaks or donor DNA},
author = {Anzalone, Andrew V. and Randolph, Peyton B. and Davis, Jessie R. and Sousa, Alexander A. and Koblan, Luke W. and Levy, Jonathan M. and Chen, Peter J. and Wilson, Christopher and Newby, Gregory A. and Raguram, Aditya and Liu, David R.},
......@@ -26,6 +26,7 @@ const bibtexSources = [
abstractnote = {Most genetic variants that contribute to disease1 are challenging to correct efficiently and without excess byproducts2–5. Here we describe prime editing, a versatile and precise genome editing method that directly writes new genetic information into a specified DNA site using a catalytically impaired Cas9 endonuclease fused to an engineered reverse transcriptase, programmed with a prime editing guide RNA (pegRNA) that both specifies the target site and encodes the desired edit. We performed more than 175 edits in human cells, including targeted insertions, deletions, and all 12 types of point mutation, without requiring double-strand breaks or donor DNA templates. We used prime editing in human cells to correct, efficiently and with few byproducts, the primary genetic causes of sickle cell disease (requiring a transversion in HBB) and Tay–Sachs disease (requiring a deletion in HEXA); to install a protective transversion in PRNP; and to insert various tags and epitopes precisely into target loci. Four human cell lines and primary post-mitotic mouse cortical neurons support prime editing with varying efficiencies. Prime editing shows higher or similar efficiency and fewer byproducts than homology-directed repair, has complementary strengths and weaknesses compared to base editing, and induces much lower off-target editing than Cas9 nuclease at known Cas9 off-target sites. Prime editing substantially expands the scope and capabilities of genome editing, and in principle could correct up to 89% of known genetic variants associated with human diseases.},
language = {en}
}`,
/* 2 -> 7 Ehemals 7, jetzt 13 */
`@article{Jinek_Chylinski_Fonfara_Hauer_Doudna_Charpentier_2012,
title = {A programmable dual RNA-guided DNA endonuclease in adaptive bacterial immunity},
author = {Jinek, Martin and Chylinski, Krzysztof and Fonfara, Ines and Hauer, Michael and Doudna, Jennifer A. and Charpentier, Emmanuelle},
......@@ -39,6 +40,7 @@ const bibtexSources = [
issn = {0036-8075},
abstractnote = {CRISPR/Cas systems provide bacteria and archaea with adaptive immunity against viruses and plasmids by using crRNAs to guide the silencing of invading nucleic acids. We show here that in a subset of these systems, the mature crRNA base-paired to trans-activating tracrRNA forms a two-RNA structure that directs the CRISPR-associated protein Cas9 to introduce double-stranded (ds) breaks in target DNA. At sites complementary to the crRNA-guide sequence, the Cas9 HNH nuclease domain cleaves the complementary strand while the Cas9 RuvC-like domain cleaves the non-complementary strand. The dual-tracrRNA:crRNA, when engineered as a single RNA chimera, also directs sequence-specific Cas9 dsDNA cleavage. Our study reveals a family of endonucleases that use dual-RNAs for site-specific DNA cleavage and highlights the potential to exploit the system for RNA-programmable genome editing., A two-RNA structure directs an endonuclease to cleave target DNA.}
}`,
/* 3 -> 8 Ehemals 8, jetzt 14 */
`@article{Gao_Ravendran_Mikkelsen_Haldrup_Cai_Ding_Paludan_Thomsen_Mikkelsen_Bak_2022,
title = {A truncated reverse transcriptase enhances prime editing by split AAV vectors},
author = {Gao, Zongliang and Ravendran, Sujan and Mikkelsen, Nanna S. and Haldrup, Jakob and Cai, Huiqiang and Ding, Xiangning and Paludan, Søren R. and Thomsen, Martin K. and Mikkelsen, Jacob Giehm and Bak, Rasmus O.},
......@@ -53,6 +55,7 @@ const bibtexSources = [
issn = {1525-0016, 1525-0024},
language = {English}
}`,
/* 4 -> 9 Hemelas 9, jetzt 15 */
`@article{Chen_Hussmann_Yan_Knipping_Ravisankar_Chen_Chen_Nelson_Newby_Sahin_2021,
title = {Enhanced prime editing systems by manipulating cellular determinants of editing outcomes},
author = {Chen, Peter J. and Hussmann, Jeffrey A. and Yan, Jun and Knipping, Friederike and Ravisankar, Purnima and Chen, Pin-Fang and Chen, Cidi and Nelson, James W. and Newby, Gregory A. and Sahin, Mustafa and Osborn, Mark J. and Weissman, Jonathan S. and Adamson, Britt and Liu, David R.},
......@@ -66,6 +69,7 @@ const bibtexSources = [
issn = {0092-8674},
abstractnote = {While prime editing enables precise sequence changes in DNA, cellular determinants of prime editing remain poorly understood. Using pooled CRISPRi screens, we discovered that DNA mismatch repair (MMR) impedes prime editing and promotes undesired indel byproducts. We developed PE4 and PE5 prime editing systems in which transient expression of an engineered MMR-inhibiting protein enhances the efficiency of substitution, small insertion, and small deletion prime edits by an average 7.7-fold and 2.0-fold compared to PE2 and PE3 systems, respectively, while improving edit/indel ratios by 3.4-fold in MMR-proficient cell types. Strategic installation of silent mutations near the intended edit can enhance prime editing outcomes by evading MMR. Prime editor protein optimization resulted in a PEmax architecture that enhances editing efficacy by 2.8-fold on average in HeLa cells. These findings enrich our understanding of prime editing and establish prime editing systems that show substantial improvement across 191 edits in seven mammalian cell types.}
}`,
/* 5 -> 10 Ehemals 10, jetzt 16 */
`@article{Tomer_Buermeyer_Nguyen_Liskay_2002,
title = {Contribution of Human Mlh1 and Pms2 ATPase Activities to DNA Mismatch Repair},
author = {Tomer, Guy and Buermeyer, Andrew B. and Nguyen, Megan M. and Liskay, R. Michael},
......@@ -79,6 +83,7 @@ const bibtexSources = [
issn = {0021-9258},
abstractnote = {MutLα, a heterodimer composed of Mlh1 and Pms2, is the major MutL activity in mammalian DNA mismatch repair. Highly conserved motifs in the N termini of both subunits predict that the protein is an ATPase. To study the significance of these motifs to mismatch repair, we have expressed in insect cells wild type human MutLα and forms altered in conserved glutamic acid residues, predicted to catalyze ATP hydrolysis of Mlh1, Pms2, or both. Using an in vitro assay, we showed that MutLα proteins altered in either glutamic acid residue were each partially defective in mismatch repair, whereas the double mutant showed no detectable mismatch repair. Neither strand specificity nor directionality of repair was affected in the single mutant proteins. Limited proteolysis studies of MutLα demonstrated that both Mlh1 and Pms2 N-terminal domains undergo ATP-induced conformational changes, but the extent of the conformational change for Mlh1 was more apparent than for Pms2. Furthermore, Mlh1 was protected at lower ATP concentrations than Pms2, suggesting Mlh1 binds ATP with higher affinity. These findings imply that ATP hydrolysis is required for MutLα activity in mismatch repair and that this activity is associated with differential conformational changes in Mlh1 and Pms2.}
}`,
/* 6 -> 11 Ehemals 11, jetzt 17 */
`@article{Wu_Corbett_Berland_2009,
title = {The Intracellular Mobility of Nuclear Import Receptors and NLS Cargoes},
author = {Wu, Jianrong and Corbett, Anita H. and Berland, Keith M.},
......@@ -92,6 +97,7 @@ const bibtexSources = [
issn = {0006-3495},
abstractnote = {We have investigated classical nuclear localization sequence (NLS) mediated protein trafficking by measuring biomolecular dynamics within living cells using two-photon fluorescence correlation spectroscopy. By directly observing the behavior of specific molecules in their native cellular environment, it is possible to uncover functional details that are not apparent from traditional biochemical investigations or functional assays. We show that the intracellular mobility of NLS cargoes and their import receptor proteins, karyopherin-α and karyopherin-β, can be robustly measured and that quantitative comparison of intracellular diffusion coefficients provides new insights into nuclear transport mechanisms. Import cargo complexes are assembled throughout the cytoplasm, and their diffusion is slower than predicted by molecular weight due to specific interactions. Analysis of NLS cargo diffusion in the cytoplasm indicates that these interactions are likely disrupted by NLS cargo binding. Our results suggest that delivery of import receptors and NLS cargoes to nuclear pores may complement selective translocation through the pores as a functional mechanism for regulating transport of proteins into the nucleus.}
}`,
/* 7 -> 12 Ehemals 12, jetzt 18 */
`@article{Dang_Lee_1988,
title = {Identification of the human c-myc protein nuclear translocation signal},
author = {Dang, C.V. and Lee, W.M.F.},
......@@ -101,7 +107,9 @@ const bibtexSources = [
number = 10,
pages = {4048–4054},
doi = {10.1128/MCB.8.10.4048}
}`,
}`
,
/* 8 -> 13 */
`@article{Spencer_Zhang_2017,
title = {Deep mutational scanning of S. pyogenes Cas9 reveals important functional domains},
author = {Spencer, J.M. and Zhang, X.},
......@@ -111,8 +119,9 @@ const bibtexSources = [
number = 1,
doi = {10.1038/s41598-017-17081-y},
abstractnote = {RNA-guided endonucleases (RGENs) have invigorated the field of site-specific nucleases. The success of Streptococcus pyogenes Cas9 (SpCas9) has led to the discovery of several other CRISPR-Associated RGENs. As more RGENs become available, it will be necessary to refine their activity before they can be translated into the clinic. With this in mind, we sought to demonstrate how deep mutational scanning (DMS) could provide details about important functional regions in SpCas9 and speed engineering efforts. Consequently, we developed a nuclease screening platform which could distinguish active Cas9 mutants. We screened a library of 1.9 × 107 with over 8500 possible non-synonymous mutations and inferred the effects of each mutation using DMS. We demonstrate that the RuvC and HNH domains are the least tolerant regions to mutation. In contrast, the Rec2 and PI domains tolerate mutation better than other regions. The mutation information defined in this work provides a foundation for further SpCas9 engineering. Together, our results demonstrate how DMS can be a powerful tool to uncover features important to RGEN function. Application of this approach to emerging RGENs should enhance their engineering and optimization for therapeutic and other applications. © 2017 The Author(s).}
}`,
`@article{Doman_Pandey_Neugebauer_An_Davis_Randolph_McElroy_Gao_Raguram_Richter_2023,
}`,
/* 9 -> 14 */
`@article{Doman_Pandey_Neugebauer_An_Davis_Randolph_McElroy_Gao_Raguram_Richter_2023,
title = {Phage-assisted evolution and protein engineering yield compact, efficient prime editors},
author = {Doman, Jordan L. and Pandey, Smriti and Neugebauer, Monica E. and An, Meirui and Davis, Jessie R. and Randolph, Peyton B. and McElroy, Amber and Gao, Xin D. and Raguram, Aditya and Richter, Michelle F. and Everette, Kelcee A. and Banskota, Samagya and Tian, Kathryn and Tao, Y. Allen and Tolar, Jakub and Osborn, Mark J. and Liu, David R.},
year = 2023,
......@@ -125,7 +134,7 @@ const bibtexSources = [
doi = {10.1016/j.cell.2023.07.039},
issn = {0092-8674, 1097-4172},
language = {English}
}`,
}`, /* 10 -> 15 */
`@article{Sousa_Hemez_Lei_Traore_Kulhankova_Newby_Doman_Oye_Pandey_Karp_2024,
title = {Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells},
author = {Sousa, Alexander A. and Hemez, Colin and Lei, Lei and Traore, Soumba and Kulhankova, Katarina and Newby, Gregory A. and Doman, Jordan L. and Oye, Keyede and Pandey, Smriti and Karp, Philip H. and McCray, Paul B. and Liu, David R.},
......@@ -137,9 +146,10 @@ const bibtexSources = [
doi = {10.1038/s41551-024-01233-3},
issn = {2157-846X},
rights = {2024 The Author(s)},
abstractnote = {Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human cystic fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE—engineered PE guide RNAs, the PEmax architecture, the transient expression of a dominant-negative mismatch repair protein, strategic silent edits, PE6 variants and proximal ‘dead’ single-guide RNAs—we increased correction efficiencies for CFTR F508del from less than 0.5% in HEK293T cells to 58% in immortalized bronchial epithelial cells (a 140-fold improvement) and to 25% in patient-derived airway epithelial cells. The optimizations also resulted in minimal off-target editing, in edit-to-indel ratios 3.5-fold greater than those achieved by nuclease-mediated homology-directed repair, and in the functional restoration of CFTR ion channels to over 50% of wild-type levels (similar to those achieved via combination treatment with elexacaftor, tezacaftor and ivacaftor) in primary airway cells. Our findings support the feasibility of a durable one-time treatment for CF.},
abstractnote = {Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human Cystic Fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE—engineered PE guide RNAs, the PEmax architecture, the transient expression of a dominant-negative mismatch repair protein, strategic silent edits, PE6 variants and proximal ‘dead’ single-guide RNAs—we increased correction efficiencies for CFTR F508del from less than 0.5% in HEK293T cells to 58% in immortalized bronchial epithelial cells (a 140-fold improvement) and to 25% in patient-derived airway epithelial cells. The optimizations also resulted in minimal off-target editing, in edit-to-indel ratios 3.5-fold greater than those achieved by nuclease-mediated homology-directed repair, and in the functional restoration of CFTR ion channels to over 50% of wild-type levels (similar to those achieved via combination treatment with elexacaftor, tezacaftor and ivacaftor) in primary airway cells. Our findings support the feasibility of a durable one-time treatment for CF.},
language = {en}
}`,
}`,
/* 11 -> 16*/
`@article{Yan_Oyler-Castrillo_Ravisankar_Ward_Levesque_Jing_Simpson_Zhao_Li_Yan_2024,
title = {Improving prime editing with an endogenous small RNA-binding protein},
author = {Yan, Jun and Oyler-Castrillo, Paul and Ravisankar, Purnima and Ward, Carl C. and Levesque, Sébastien and Jing, Yangwode and Simpson, Danny and Zhao, Anqi and Li, Hui and Yan, Weihao and Goudy, Laine and Schmidt, Ralf and Solley, Sabrina C. and Gilbert, Luke A. and Chan, Michelle M. and Bauer, Daniel E. and Marson, Alexander and Parsons, Lance R. and Adamson, Britt},
......@@ -153,7 +163,9 @@ const bibtexSources = [
issn = {0028-0836, 1476-4687},
abstractnote = {Abstract Prime editing enables the precise modification of genomes through reverse transcription of template sequences appended to the 3′ ends of CRISPR–Cas guide RNAs 1 . To identify cellular determinants of prime editing, we developed scalable prime editing reporters and performed genome-scale CRISPR-interference screens. From these screens, a single factor emerged as the strongest mediator of prime editing: the small RNA-binding exonuclease protection factor La. Further investigation revealed that La promotes prime editing across approaches (PE2, PE3, PE4 and PE5), edit types (substitutions, insertions and deletions), endogenous loci and cell types but has no consistent effect on genome-editing approaches that rely on standard, unextended guide RNAs. Previous work has shown that La binds polyuridine tracts at the 3′ ends of RNA polymerase III transcripts 2 . We found that La functionally interacts with the 3′ ends of polyuridylated prime editing guide RNAs (pegRNAs). Guided by these results, we developed a prime editor protein (PE7) fused to the RNA-binding, N-terminal domain of La. This editor improved prime editing with expressed pegRNAs and engineered pegRNAs (epegRNAs), as well as with synthetic pegRNAs optimized for La binding. Together, our results provide key insights into how prime editing components interact with the cellular environment and suggest general strategies for stabilizing exogenous small RNAs therein.},
language = {en}
}`,
}`,
/* 12 -> 17*/
`@article{Simon,
title = {PEAR, a flexible fluorescent reporter for the identification and enrichment of successfully prime edited cells},
author = {Simon, Dorottya Anna and Tálas, András and Kulcsár, Péter István and Biczók, Zsuzsanna and Krausz, Sarah Laura and Várady, György and Welker, Ervin},
......@@ -166,5 +178,5 @@ const bibtexSources = [
issn = {2050-084X},
abstractnote = {Prime editing is a recently developed CRISPR/Cas9 based gene engineering tool that allows the introduction of short insertions, deletions, and substitutions into the genome. However, the efficiency of prime editing, which typically achieves editing rates of around 10%–30%, has not matched its versatility. Here, we introduce the prime editor activity reporter (PEAR), a sensitive fluorescent tool for identifying single cells with prime editing activity. PEAR has no background fluorescence and specifically indicates prime editing events. Its design provides apparently unlimited flexibility for sequence variation along the entire length of the spacer sequence, making it uniquely suited for systematic investigation of sequence features that influence prime editing activity. The use of PEAR as an enrichment marker for prime editing can increase the edited population by up to 84%, thus significantly improving the applicability of prime editing for basic research and biotechnological applications.},
editor = {Lapinaite, Audrone and Stainier, Didier YR and Hamilton, Jennifer R}
}`
}`
]
\ No newline at end of file
src/sources/eng-peg-sources.tsx
View file @ 3fd292fd
......@@ -3,7 +3,7 @@ import BibtexParser from "../components/makeSources";
export default function EngPegsources(){
return (
<div>
<BibtexParser bibtexSources={bibtexSources} />
<BibtexParser bibtexSources={bibtexSources} start={18} />
</div>
);
}
......@@ -65,12 +65,12 @@ const bibtexSources = [
doi = {10.1016/S0014-5793(01)02561-3},
issn = {1873-3468},
rights = {FEBS Letters 499 (2001) 1873-3468 © 2015 Federation of European Biochemical Societies},
abstractnote = {The green fluorescent protein YFP-H148Q is sensitive to halides by a mechanism involving halide binding and a shift in pK a. However, a limitation of YFP-H148Q is its low halide sensitivity, with K d>100 mM for Cl−. Indicators with improved sensitivities are needed for cell transport studies, particularly in drug discovery by high-throughput screening, and for measurement of Cl− concentration in subcellular organelles. YFP-H148Q libraries were generated in which pairs of residues in the vicinity of the halide binding site were randomly mutated. An automated procedure was developed to screen bacterial colonies for improved halide sensitivity. Analysis of 1536 clones revealed improved anion sensitivities with K d down to 2 mM for I− (I152L), 40 mM for Cl− (V163S), and 10 mM for NO3 − (I152L). The anion-sensitive mechanism of these indicators was established and their utility in cells was demonstrated using transfected cells expressing the cystic fibrosis transmembrane conductance regulator chloride channel.},
abstractnote = {The green fluorescent protein YFP-H148Q is sensitive to halides by a mechanism involving halide binding and a shift in pK a. However, a limitation of YFP-H148Q is its low halide sensitivity, with K d>100 mM for Cl−. Indicators with improved sensitivities are needed for cell transport studies, particularly in drug discovery by high-throughput screening, and for measurement of Cl− concentration in subcellular organelles. YFP-H148Q libraries were generated in which pairs of residues in the vicinity of the halide binding site were randomly mutated. An automated procedure was developed to screen bacterial colonies for improved halide sensitivity. Analysis of 1536 clones revealed improved anion sensitivities with K d down to 2 mM for I− (I152L), 40 mM for Cl− (V163S), and 10 mM for NO3 − (I152L). The anion-sensitive mechanism of these indicators was established and their utility in cells was demonstrated using transfected cells expressing the Cystic Fibrosis transmembrane conductance regulator chloride channel.},
language = {en}
}
`,`
@article{Bulcaen_Kortleven_Liu_Maule_Dreano_Kelly_Ensinck_Thierie_Smits_Ciciani_2024,
title = {Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
title = {Prime editing functionally corrects Cystic Fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
author = {Bulcaen, Mattijs and Kortleven, Phéline and Liu, Ronald B. and Maule, Giulia and Dreano, Elise and Kelly, Mairead and Ensinck, Marjolein M. and Thierie, Sam and Smits, Maxime and Ciciani, Matteo and Hatton, Aurelie and Chevalier, Benoit and Ramalho, Anabela S. and Casadevall i Solvas, Xavier and Debyser, Zeger and Vermeulen, François and Gijsbers, Rik and Sermet-Gaudelus, Isabelle and Cereseto, Anna and Carlon, Marianne S.},
year = 2024,
month = may,
......@@ -78,7 +78,7 @@ const bibtexSources = [
pages = 101544,
doi = {10.1016/j.xcrm.2024.101544},
issn = {2666-3791},
abstractnote = {Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause cystic fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines. These findings were subsequently validated in patient-derived rectal organoids and human nasal epithelial cells. Through analysis of predicted and experimentally identified candidate off-target sites in primary stem cells, we confirm previous reports on the high prime editor (PE) specificity and its potential for a curative CF gene editing therapy. To facilitate future screening of genetic strategies in a translational CF model, a machine learning algorithm was developed for dynamic quantification of CFTR function in organoids (DETECTOR: “detection of targeted editing of CFTR in organoids”).}
abstractnote = {Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause Cystic Fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines. These findings were subsequently validated in patient-derived rectal organoids and human nasal epithelial cells. Through analysis of predicted and experimentally identified candidate off-target sites in primary stem cells, we confirm previous reports on the high prime editor (PE) specificity and its potential for a curative CF gene editing therapy. To facilitate future screening of genetic strategies in a translational CF model, a machine learning algorithm was developed for dynamic quantification of CFTR function in organoids (DETECTOR: “detection of targeted editing of CFTR in organoids”).}
}
`,`
@article{Renz_Valdivia-Francia_Sendoel_2020,
......
src/sources/eng-trf-sources.tsx
View file @ 3fd292fd
......@@ -3,7 +3,7 @@ import BibtexParser from "../components/makeSources";
export default function EngTrfsources(){
return (
<div>
<BibtexParser bibtexSources={bibtexSources} />
<BibtexParser bibtexSources={bibtexSources} start={3} />
</div>
);
}
......
src/sources/entre-sources.tsx 0 → 100644
View file @ 3fd292fd
import BibtexParser from "../components/makeSources";
export default function EntrepreneurSources(){
return (
<div>
<BibtexParser bibtexSources={bibtexSources} special="ent"/>
</div>
);
}
const bibtexSources = [
`
@article{GMI2021CysticFibrosis,
title = {Cystic Fibrosis Therapeutics Market Size - By Treatment Method (Medications [Drug Class {CFTR Modulators, Mucolytics, Bronchodilators, Pancreatic Enzyme Supplements}, Route of Administration], Devices), Distribution Channel & Forecast, 2021 - 2027},
author = {Faizullabhoy, Mariam and Wani, Gauri},
journal = {GMI Insights},
year = {2021},
month = {Sep},
url = {https://www.gminsights.com/industry-analysis/cystic-fibrosis-therapeutics-market},
note = {Report ID: GMI5118}
}
`,
`
@article{Fajac2021,
title={Therapeutic Approaches for Patients with Cystic Fibrosis Not Eligible for Current CFTR Modulators},
author={Fajac, Isabelle and Sermet, Isabelle},
journal={Cells},
volume={10},
number={10},
year={2021},
month={Oct},
doi={10.3390/cells10102793},
url={https://pubmed.ncbi.nlm.nih.gov/34685773/},
}
`,
`
@misc{ExpertMarketResearch2023,
title = {Cystic Fibrosis Treatment Market Report and Forecast 2024-2032},
author = {Rahul Gotadki},
year = {2023},
month = {Feb},
url = {https://www.expertmarketresearch.com/reports/cystic-fibrosis-treatment-market},
note = {ID: MRFR/Pharma/1293-CR}
}
`,
`
@misc{DeWeerdt2016,
author = {Sarah DeWeerdt},
title = {Developing gene therapy to treat cystic fibrosis: challenges and successes},
journal = {The Pharmaceutical Journal},
year = {2016},
month = {Jun},
url = {https://pharmaceutical-journal.com/article/feature/developing-gene-therapy-to-treat-cystic-fibrosis-challenges-and-successes},
note = {Accessed: 30 Sep 2024}
}
`
]
\ No newline at end of file
src/sources/joshua-inv-sources.tsx
View file @ 3fd292fd
......@@ -14,7 +14,7 @@ const bibtexSources = [
@article{eins,
title = {Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
title = {Prime editing functionally corrects Cystic Fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
journal = {Cell Reports Medicine},
......@@ -45,7 +45,7 @@ month = {11},
pages = {1756-61},
title = {Cystic fibrosis in three northeast Thai infants is CF really a rare disease in the Thai population?},
title = {Cystic Fibrosis in three northeast Thai infants is CF really a rare disease in the Thai population?},
volume = {89},
......@@ -63,7 +63,7 @@ month = {01},
pages = {8},
title = {Cystic fibrosis in asia},
title = {Cystic Fibrosis in asia},
volume = {4},
......
src/sources/liu-inv-sources.tsx
View file @ 3fd292fd
......@@ -64,7 +64,7 @@ title = {Systematic optimization of prime editing for the efficient functional c
rights = {2024 The Author(s)},
ISSN = {2157-846X},
DOI = {10.1038/s41551-024-01233-3},
abstractNote = {Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human cystic fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE—engineered PE guide RNAs, the PEmax architecture, the transient expression of a dominant-negative mismatch repair protein, strategic silent edits, PE6 variants and proximal ‘dead’ single-guide RNAs—we increased correction efficiencies for CFTR F508del from less than 0.5% in HEK293T cells to 58% in immortalized bronchial epithelial cells (a 140-fold improvement) and to 25% in patient-derived airway epithelial cells. The optimizations also resulted in minimal off-target editing, in edit-to-indel ratios 3.5-fold greater than those achieved by nuclease-mediated homology-directed repair, and in the functional restoration of CFTR ion channels to over 50% of wild-type levels (similar to those achieved via combination treatment with elexacaftor, tezacaftor and ivacaftor) in primary airway cells. Our findings support the feasibility of a durable one-time treatment for CF.},
abstractNote = {Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human Cystic Fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE—engineered PE guide RNAs, the PEmax architecture, the transient expression of a dominant-negative mismatch repair protein, strategic silent edits, PE6 variants and proximal ‘dead’ single-guide RNAs—we increased correction efficiencies for CFTR F508del from less than 0.5% in HEK293T cells to 58% in immortalized bronchial epithelial cells (a 140-fold improvement) and to 25% in patient-derived airway epithelial cells. The optimizations also resulted in minimal off-target editing, in edit-to-indel ratios 3.5-fold greater than those achieved by nuclease-mediated homology-directed repair, and in the functional restoration of CFTR ion channels to over 50% of wild-type levels (similar to those achieved via combination treatment with elexacaftor, tezacaftor and ivacaftor) in primary airway cells. Our findings support the feasibility of a durable one-time treatment for CF.},
journal = {Nature Biomedical Engineering},
publisher = {Nature Publishing Group},
author = {Sousa, Alexander A. and Hemez, Colin and Lei, Lei and Traore, Soumba and Kulhankova, Katarina and Newby, Gregory A. and Doman, Jordan L. and Oye, Keyede and Pandey, Smriti and Karp, Philip H. and McCray, Paul B. and Liu, David R.},
......
src/sources/mattij-inv-sources.tsx
View file @ 3fd292fd
......@@ -13,7 +13,7 @@ const bibtexSources = [
`
@article{Bulcaen_Kortleven_Liu_Maule_Dreano_Kelly_Ensinck_Thierie_Smits_Ciciani_et,
title = {
Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in
Prime editing functionally corrects Cystic Fibrosis-causing CFTR mutations in
human organoids and airway epithelial cells
},
author = {
......@@ -34,7 +34,7 @@ const bibtexSources = [
Prime editing is a recent, CRISPR-derived genome editing technology capable
of introducing precise nucleotide substitutions, insertions, and deletions.
Here, we present prime editing approaches to correct L227R- and N1303K-CFTR,
two mutations that cause cystic fibrosis and are not eligible for current
two mutations that cause Cystic Fibrosis and are not eligible for current
market-approved modulator therapies. We show that, upon DNA correction of the
CFTR gene, the complex glycosylation, localization, and, most importantly,
function of the CFTR protein are restored in HEK293T and 16HBE cell lines.
......
src/sources/methods-sources.tsx
View file @ 3fd292fd
......@@ -171,7 +171,7 @@ const bibtexSources = [
`,
`
@article{BULCAEN2024101544,
title = {Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
title = {Prime editing functionally corrects Cystic Fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
journal = {Cell Reports Medicine},
volume = {5},
number = {5},
......@@ -181,9 +181,9 @@ issn = {2666-3791},
doi = {https://doi.org/10.1016/j.xcrm.2024.101544},
url = {https://www.sciencedirect.com/science/article/pii/S2666379124002349},
author = {Mattijs Bulcaen and Phéline Kortleven and Ronald B. Liu and Giulia Maule and Elise Dreano and Mairead Kelly and Marjolein M. Ensinck and Sam Thierie and Maxime Smits and Matteo Ciciani and Aurelie Hatton and Benoit Chevalier and Anabela S. Ramalho and Xavier {Casadevall i Solvas} and Zeger Debyser and François Vermeulen and Rik Gijsbers and Isabelle Sermet-Gaudelus and Anna Cereseto and Marianne S. Carlon},
keywords = {cystic fibrosis, prime editing, patient-derived organoids, human nasal epithelial cells, gene editing, machine learning, DETEOR, CRISPR},
keywords = { Cystic Fibrosis, prime editing, patient-derived organoids, human nasal epithelial cells, gene editing, machine learning, DETEOR, CRISPR},
abstract = {Summary
Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause cystic fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines. These findings were subsequently validated in patient-derived rectal organoids and human nasal epithelial cells. Through analysis of predicted and experimentally identified candidate off-target sites in primary stem cells, we confirm previous reports on the high prime editor (PE) specificity and its potential for a curative CF gene editing therapy. To facilitate future screening of genetic strategies in a translational CF model, a machine learning algorithm was developed for dynamic quantification of CFTR function in organoids (DETECTOR: “detection of targeted editing of CFTR in organoids”).}
Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause Cystic Fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines. These findings were subsequently validated in patient-derived rectal organoids and human nasal epithelial cells. Through analysis of predicted and experimentally identified candidate off-target sites in primary stem cells, we confirm previous reports on the high prime editor (PE) specificity and its potential for a curative CF gene editing therapy. To facilitate future screening of genetic strategies in a translational CF model, a machine learning algorithm was developed for dynamic quantification of CFTR function in organoids (DETECTOR: “detection of targeted editing of CFTR in organoids”).}
}
new8.
@article{Ensinck_Deeersmaecker_Heylen_Ramalho_Gijsbers_Far,
......@@ -238,8 +238,8 @@ new8.
`,
`@article{ehrhardt_towards_2006,
title = {Towards an in vitro model of cystic fibrosis small airway epithelium: characterisation of the human bronchial epithelial cell line {CFBE41o}-},
shorttitle = {Towards an in vitro model of cystic fibrosis small airway epithelium},
title = {Towards an in vitro model of Cystic Fibrosis small airway epithelium: characterisation of the human bronchial epithelial cell line {CFBE41o}-},
shorttitle = {Towards an in vitro model of Cystic Fibrosis small airway epithelium},
author = {Ehrhardt, Carsten and Collnot, Eva-Maria and Baldes, Christiane and Becker, Ulrich and Laue, Michael and Kim, Kwang-Jin and Lehr, Claus-Michael},
year = 2006,
month = mar,
......@@ -253,7 +253,7 @@ new8.
urldate = {2024-09-09},
copyright = {http://www.springer.com/tdm},
language = {en},
file = {Ehrhardt et al. - 2006 - Towards an in vitro model of cystic fibrosis small.pdf:C\:\\Users\\Isabell\\Zotero\\storage\\RXLMCE3V\\Ehrhardt et al. - 2006 - Towards an in vitro model of cystic fibrosis small.pdf:application/pdf}
file = {Ehrhardt et al. - 2006 - Towards an in vitro model of Cystic Fibrosis small.pdf:C\:\\Users\\Isabell\\Zotero\\storage\\RXLMCE3V\\Ehrhardt et al. - 2006 - Towards an in vitro model of Cystic Fibrosis small.pdf:application/pdf}
}
`
,
......
src/sources/part-sources.tsx 0 → 100644
View file @ 3fd292fd
import BibtexParser from "../components/makeSources";
export default function PartSources(){
return (
<div>
<BibtexParser bibtexSources={bibtexSources} />
</div>
);
}
const bibtexSources = [
`
@article {10.7554/eLife.69504,
article_type = {journal},
title = {PEAR, a flexible fluorescent reporter for the identification and enrichment of successfully prime edited cells},
author = {Simon, Dorottya Anna and Tálas, András and Kulcsár, Péter István and Biczók, Zsuzsanna and Krausz, Sarah Laura and Várady, György and Welker, Ervin},
editor = {Lapinaite, Audrone and Stainier, Didier YR and Hamilton, Jennifer R},
volume = 11,
year = 2022,
month = {feb},
pub_date = {2022-02-23},
pages = {e69504},
citation = {eLife 2022;11:e69504},
doi = {10.7554/eLife.69504},
url = {https://doi.org/10.7554/eLife.69504},
abstract = {Prime editing is a recently developed CRISPR/Cas9 based gene engineering tool that allows the introduction of short insertions, deletions, and substitutions into the genome. However, the efficiency of prime editing, which typically achieves editing rates of around 10\%–30\%, has not matched its versatility. Here, we introduce the prime editor activity reporter (PEAR), a sensitive fluorescent tool for identifying single cells with prime editing activity. PEAR has no background fluorescence and specifically indicates prime editing events. Its design provides apparently unlimited flexibility for sequence variation along the entire length of the spacer sequence, making it uniquely suited for systematic investigation of sequence features that influence prime editing activity. The use of PEAR as an enrichment marker for prime editing can increase the edited population by up to 84\%, thus significantly improving the applicability of prime editing for basic research and biotechnological applications.},
keywords = {CRISPR, Cas9, prime editing, fluorescent, fluorescent reporter, reporter, enrichment, genome engineering},
journal = {eLife},
issn = {2050-084X},
publisher = {eLife Sciences Publications, Ltd},
}
`,
`
@misc{nickaseassay2024internet,
author = {Kim et al.},
title = { Utilization of nicking properties of CRISPR-Cas12a effector for genome editing},
year = {2024},
url = { https://doi.org/10.1038/s41598-024-53648-2},
note = {Zugriff am 16. August 2024}
}
`
]
\ No newline at end of file
src/sources/res-sources.tsx 0 → 100644
View file @ 3fd292fd
import BibtexParser from "../components/makeSources";
export default function ResultSources(){
return (
<div>
<BibtexParser bibtexSources={bibtexSources} />
</div>
);
}
const bibtexSources = [
`
@article{wang2023sortlnps,
author = {Wang, X. and Liu, S. and Sun, Y. and others},
title = {Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs) using multiple technical methods for tissue-specific mRNA delivery},
journal = {Nature Protocols},
volume = {18},
pages = {265--291},
year = {2023},
doi = {10.1038/s41596-022-00755-},
url = {https://doi.org/10.1038/s41596-022-00755-}
}
`,
`@article{sousa2024primeediting,
author = {Sousa, A. A. and Hemez, C. and Lei, L. and others},
title = {Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells},
journal = {Nature Biomedical Engineering},
year = {2024},
doi = {10.1038/s41551-024-01233-3},
url = {https://doi.org/10.1038/s41551-024-01233-3}
}
`
]
\ No newline at end of file