Article

A systemically delivered AAV-CFTR gene therapy for cystic fibrosis

2025-03-20

Abstract excerpt

Cystic fibrosis (CF) is the most common monogenic lung disease and results from mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). There have been over 2000 variants identified in patients that result in loss of function of the CFTR protein leading to systemic disease and respiratory failure in adolescence. While some variants encode proteins with residual activity that can be corrected...

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Literature Corpus work: 4600d924-7190-5d83-b6d7-04c0abe32ea1
DOI: 10.1101/2025.03.20.642115

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