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CFF VLC 2022

33 - Navigating the Path: Approaches and Progress Toward Genetic Therapies for CF

‐ Jul 24, 2022 1:30pm

Understand the basic biology behind cystic fibrosis and the approaches to treat the underlying cause of the disease – mutations in the CF transmembrane regulator (CFTR) gene. Learn how mutations in different parts of the gene result in diverse protein defects, and in some cases complete loss of CFTR protein. This session will also break down the most common approaches for genetic therapies, which aim to restore functional CFTR expression by delivering RNA, DNA, or DNA editing machinery. If successful, these therapies could benefit all people with CF, including those with rare or nonsense mutations. To close the session, you will hear about the challenge of packaging and delivering genetic therapies for CF and learn how the Foundation is working to overcome this hurdle.


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