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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...
Autores principales: | Farinha, Carlos M, Sousa, Marisa, Canato, Sara, Schmidt, André, Uliyakina, Inna, Amaral, Margarida D |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons, Ltd
2015
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Materias: | |
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 |
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