S945L-CFTR molecular dynamics, functional characterization and tezacaftor/ivacaftor efficacy in vivo and in vitro in matched pediatric patient-derived cell models

• Main Authors: Katelin M. Allan (Author), Miro A. Astore (Author), Laura K. Fawcett (Author), Sharon L. Wong (Author), Po-Chia Chen (Author), Renate Griffith (Author), Adam Jaffe (Author), Serdar Kuyucak (Author), Shafagh A. Waters (Author)
• Format: Book
• Published: Frontiers Media S.A., 2022-11-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Cystic Fibrosis (CF) results from over 400 different disease-causing mutations in the CF Transmembrane Conductance Regulator (CFTR) gene. These CFTR mutations lead to numerous defects in CFTR protein function. A novel class of targeted therapies (CFTR modulators) have been developed that can restore...