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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Bibliografiska uppgifter
I publikationen:Pharmacol Res Perspect
Huvudupphovsmän: Farinha, Carlos M, Sousa, Marisa, Canato, Sara, Schmidt, André, Uliyakina, Inna, Amaral, Margarida D
Materialtyp: Artigo
Språk:Inglês
Publicerad: John Wiley & Sons, Ltd 2015
Ämnen:
Original Articles
Länkar: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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