<QaBoxq="Are most of the other patients you know in good health like you?"a="No. Another boy my age got a fungal infection and does not have long time left to live. "/>
</>,
summary:"We reached out to Max, a friend of a teammate, to gain insights into the needs and experiences of cystic fibrosis (CF) patients. Our discussions revealed the challenges faced by CF patients, even those in relatively good health, and emphasized the ongoing need for new treatment options. Max's candid sharing of his experiences highlighted the limitations of current modulators, the importance of lung function, and the impact of treatments on quality of life. This meeting transformed our project perspective, urging us to prioritize safety and real-world benefits in our design. Ultimately, Max's influence led us to focus on lung-targeted gene therapy instead of a diagnostic approach, reinforcing our commitment to Integrated Human Practices.",
months:"April"
months:"April",
/*interview:<iframe title="Bielefeld-CeBiTec: Interview with Max Beckmann (2024) [English]" width="560" height="315" src="https://video.igem.org/videos/embed/16501867-a687-4205-949a-51ead876e109" frameborder="0" allowfullscreen="" sandbox="allow-same-origin allow-scripts allow-popups allow-forms"></iframe>,*/
title = {Search-and-replace genome editing without double-strand breaks or donor DNA},
title = {Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
volume = {576},
journal = {Cell Reports Medicine},
rights = {2019 The Author(s), under exclusive licence to Springer Nature Limited},
volume = {5},
ISSN = {1476-4687},
number = {5},
DOI = {10.1038/s41586-019-1711-4},
pages = {101544},
abstractNote = {Most genetic variants that contribute to disease are challenging to correct efficiently and without excess byproducts. 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 and Tay–Sachs disease; 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.},
year = {2024},
number = {7785},
issn = {2666-3791},
journal = {Nature},
doi = {https://doi.org/10.1016/j.xcrm.2024.101544},
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.},
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},}
