Article
Considerations for early life genetic therapies in cystic fibrosis.
American journal of physiology. Lung cellular and molecular physiology - 1 Jun 2026
Cooney Ashley L, Thornell Ian M, Pezzulo Alejandro A, Fischer Anthony J, Cook Daniel P, Abou Alaiwa Mahmoud H, Larson Ode Katie M, Uc Aliye, Zabner Joseph, Ryan Amy L, Stoltz David A, Sinn Patrick L, McCray Paul B, Meyerholz David K
Abstract excerpt
Cystic fibrosis (CF) is caused by mutations in the gene encoding for the CF transmembrane conductance regulator (CFTR) anion channel. Since the initial characterization of CF in the early 20th century, advances in clinical management have reduced disease burden and increased longevity. These advances are amplified by the recent development of highly effective CFTR modulator therapies (HEMTs) that have provided...
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