Gene Therapy for Cystic Fibrosis Lung Disease: Overcoming the Barriers to Translation to the Clinic

• Autori principali: Martin Donnelley (Autore), David W. Parsons (Autore)
• Natura: Libro
• Pubblicazione: Frontiers Media S.A., 2018-11-01T00:00:00Z.
• Soggetti: article
• Accesso online: Connect to this object online.

Cystic fibrosis (CF) is a progressive, chronic and debilitating genetic disease caused by mutations in the CF Transmembrane-Conductance Regulator (CFTR) gene. Unrelenting airway disease begins in infancy and produces a steady deterioration in quality of life, ultimately leading to premature death. W...