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Article

Targeting F508del-CFTR to develop rational new therapies for cystic fibrosis.

Acta pharmacologica Sinica - 1 Jun 2011

Cai Zhi-wei, Liu Jia, Li Hong-yu, Sheppard David N

Abstract excerpt

The mutation F508del is the commonest cause of the genetic disease cystic fibrosis (CF). CF disrupts the function of many organs in the body, most notably the lungs, by perturbing salt and water transport across epithelial surfaces. F508del causes harm in two principal ways. First, the mutation prevents delivery of the cystic fibrosis transmembrane conductance regulator (CFTR) to its correct cellular location,...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 21642944
DOI: 10.1038/aps.2011.71
PMCID: PMC4009972

Genes

CFTR

Provenance

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