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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Detalles Bibliográficos
Autores principales: Allan, Katelin M., Astore, Miro A., Fawcett, Laura K., Wong, Sharon L., Chen, Po-Chia, Griffith, Renate, Jaffe, Adam, Kuyucak, Serdar, Waters, Shafagh A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Pediatrics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9709344/
https://www.ncbi.nlm.nih.gov/pubmed/36467478
http://dx.doi.org/10.3389/fped.2022.1062766

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9709344/
https://www.ncbi.nlm.nih.gov/pubmed/36467478
http://dx.doi.org/10.3389/fped.2022.1062766

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