American journal of respiratory and critical care medicine
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Am. J. Respir. Crit. Care Med. · Dec 2024
ReviewLentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-In-Human Trial.
Cystic fibrosis (CF) is a genetic disease caused by mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. Although CF is a multiorgan disease, the leading causes of morbidity and mortality are related to progressive lung disease. Current understanding of the effects of the broad spectrum of CFTR mutations on CFTR function has allowed for the development of CFTR modulator therapies. ⋯ A third-generation lentiviral vector pseudotyped with Sendai virus F and HN envelope proteins (rSIV. F/HN) has been developed for the treatment of CF. Promising preclinical results support the progression of this vector carrying a full-length CFTR transgene (BI 3720931) into a first-in-human clinical trial expected to begin in 2024.