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

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

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...

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Bibliographic Details
Main Authors: Katelin M. Allan, Miro A. Astore, Laura K. Fawcett, Sharon L. Wong, Po-Chia Chen, Renate Griffith, Adam Jaffe, Serdar Kuyucak, Shafagh A. Waters
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-11-01
Series:Frontiers in Pediatrics
Subjects:
cystic fibrosis
CFTR
modulators
airway epithelial cell models
personalized medicine
molecular dynamics
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2022.1062766/full

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