<pdata-aos="zoom-y-out">Cystic Fibrosis (CF) is a common life-limiting genetic disorder, particularly affecting the Caucasian population, with approximately <b>162,400 people worldwide</b> living with the condition<SupScrollLinklabel="4"/>{/* ehem7 */}. Statistically, about <b>one in every 3,300</b> white newborns is born with CF <SupScrollLinklabel="5"/>{/*ehem8*/} . And according to the German Cystic Fibrosis Registry, the average life expectancy for children born with CF in 2021 was around 57 years <SupScrollLinklabel="9"/> , highlighting the severe and life-shortening nature of the disease. </p>
<pdata-aos="zoom-y-out">Cystic Fibrosis (CF) is a common life-limiting genetic disorder, particularly affecting the Caucasian population, with approximately <b>162,400 people worldwide</b> living with the condition<SupScrollLinklabel="4"/>{/* ehem7 */}. Statistically, about <b>one in every 3,300</b> white newborns is born with CF <SupScrollLinklabel="5"/>{/*ehem8*/} . And according to the German Cystic Fibrosis Registry, the average life expectancy for children born with CF in 2021 was around 57 years <SupScrollLinklabel="6"/>{/*ehem9*/} , highlighting the severe and life-shortening nature of the disease. </p>
<p>The modern understanding of CF dates back to 1922 when Dr. Dorothy Andersen, a pediatric specialist, first described the disease and coined the term "Cystic Fibrosis"<SupScrollLinklabel="10"/>. In Germany, it is commonly known as "Mukoviszidose," derived from the Latin words meaning "mucus" and "viscous" <SupScrollLinklabel="10"/> , emphasizing the characteristic thick, sticky mucus that defines the condition <SupScrollLinklabel="11"/><sup>,</sup><SupScrollLinklabel="12"/>. </p>
<p>Genetic research has identified over 1,700 mutations in the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, with the F508del mutation being the most common, affecting about 70% of CF patients. This mutation prevents the proper folding of the CFTR protein, significantly impairing its function <SupScrollLinklabel="13"/> . </p>
<p>The CFTR protein regulates the flow of chloride ions across the membranes of cells in the lungs, digestive system, and other organs. This ion flow is essential for drawing water into surrounding tissues, which helps maintain the proper hydration and consistency of mucus. In patients with CF, the disruption of this process prevents sufficient water from entering the mucus, making it abnormally thick and sticky. The accumulation of this mucus leads to an obstruction of airways and digestive ducts, resulting in chronic lung infections, inflammation, impaired digestion, and malnutrition<SupScrollLinklabel="14"/> . </p>
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<p>Lipid nanoparticles, short LNPs, are small, spherical structures made of lipids that serve as delivery vehicles for therapeutic molecules, such as RNA, DNA, or drugs. They protect their cargo from degradation, enhance cellular uptake, and are widely used in mRNA vaccines and gene therapy due to their efficiency and biocompatibility.</p>
<H4text="LNPs and their impact on modern medicine"id="text"/>
<p>LNPs are an advanced delivery system designed to transport therapeutic molecules like RNA, DNA or proteins into the cells. These nanoparticles are tiny spheres made of lipids that form a protective shell around the cargo. The size of LNPs typically ranges from 50 to 200 nm in diameter, making them incredibly small - about 1,000 times thinner than a human hair <SupScrollLinklabel="1"/>. </p>
<p>Overall, LNPs represent a significant advancement in drug delivery technology. LNPs offer exceptionally high drug-loading capacities, making them highly effective for delivering substantial amounts of therapeutic agents in a single dose. Their advanced design allows for the encapsulation of a large payload, which enhances the efficacy of treatments and reduces the frequency of administration. By encapsulating and protecting therapeutic agents like mRNA, LNPs enhance the stability, targeted delivery, and effectiveness of treatments. Their ability to be tailored for specific delivery needs, such as targeting particular organs or overcoming physiological barriers, makes them a powerful tool in modern medicine <SupScrollLinklabel="9"/> .</p>
<p>Overall, LNPs represent a significant advancement in drug delivery technology. LNPs offer exceptionally high drug-loading capacities, making them highly effective for delivering substantial amounts of therapeutic agents in a single dose. Their advanced design allows for the encapsulation of a large payload, which enhances the efficacy of treatments and reduces the frequency of administration. By encapsulating and protecting therapeutic agents like mRNA, LNPs enhance the stability, targeted delivery, and effectiveness of treatments. Their ability to be tailored for specific delivery needs, such as targeting particular organs or overcoming physiological barriers, makes them a powerful tool in modern medicine <SupScrollLinklabel="6"/>{/*ehem9*/} .</p>
<H4text="Protection of cargo"id="text"/>
<p> The primary function of LNPs is to shield the therapeutic agents they carry, such as mRNA, from degradation and facilitate their delivery into cells. mRNA is a critical component in many modern vaccines and therapies, but it is highly susceptible to breaking down before it can reach its target within cells. LNPs address this challenge by encapsulating the mRNA, thus protecting it from harmful enzymes, like RNases and environmental conditions <SupScrollLinklabel="2"/> . </p>
