Gene Therapy for Cystic Fibrosis: Lessons Learned and Paths Forward

Abstract excerpt

Cystic fibrosis (CF) is an autosomal recessive disease caused by defects in an anion channel known as the CF transmembrane conductance regulator (CFTR). Although CF is a multi-organ disease, the primary cause of patient mortality is chronic bacterial infections of the lung, and thus this organ has been the primary target of gene therapy. For 90% of patients, small-molecule drugs that correct the basic defects in...

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