Increased efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR

Cystic fibrosis (CF), the most common recessive autosomal disease among Caucasians, is caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. The most common mutation, F508del, leads to CFTR impaired plasma membrane trafficking. Therapies modulating CFTR...

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Detalhes bibliográficos
Publicado no:Pharmacol Res Perspect
Main Authors: Farinha, Carlos M, Sousa, Marisa, Canato, Sara, Schmidt, André, Uliyakina, Inna, Amaral, Margarida D
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley & Sons, Ltd 2015
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4492728/
https://ncbi.nlm.nih.gov/pubmed/26171232
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/prp2.152
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