Gene Therapy: A Possible Alternative to CFTR Modulators?

• Prif Awduron: J. Mercier (Awdur), M. Ruffin (Awdur), H. Corvol (Awdur), L. Guillot (Awdur)
• Fformat: Llyfr
• Cyhoeddwyd: Frontiers Media S.A., 2021-04-01T00:00:00Z.
• Pynciau: article
• Mynediad Ar-lein: Connect to this object online.

Cystic fibrosis (CF) is a rare genetic disease that affects several organs, but lung disease is the major cause of morbidity and mortality. The gene responsible for CF, the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, has been discovered in 1989. Since then, gene therapy i.e., de...