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...

Descripción completa

Detalles Bibliográficos
Autores principales: Farinha, Carlos M, Sousa, Marisa, Canato, Sara, Schmidt, André, Uliyakina, Inna, Amaral, Margarida D
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Ltd 2015
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4492728/
https://www.ncbi.nlm.nih.gov/pubmed/26171232
http://dx.doi.org/10.1002/prp2.152

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4492728/
https://www.ncbi.nlm.nih.gov/pubmed/26171232
http://dx.doi.org/10.1002/prp2.152

Ejemplares similares