Therapeutic approaches to CFTR dysfunction: From discovery to drug development.

doi:10.1016/j.jcf.2017.08.013

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Article

Therapeutic approaches to CFTR dysfunction: From discovery to drug development.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Mar 2018

Li Hongyu, Pesce Emanuela, Sheppard David N, Singh Ashvani K, Pedemonte Nicoletta

Abstract excerpt

Cystic fibrosis (CF) mutations have complex effects on the cystic fibrosis transmembrane conductance regulator (CFTR) protein. They disrupt its processing to and stability at the plasma membrane and function as an ATP-gated Cl- channel. Here, we review therapeutic strategies to overcome defective CFTR processing and stability. Because CF mutations have multiple impacts on the assembly of CFTR protein, combination...

Topics

Cystic Fibrosis
Cystic Fibrosis Transmembrane Conductance Regulator
Drug Development
Humans
Ion Transport
Mutation

Source

Open in PubMed

Identifiers

PMID: 28916430
DOI: 10.1016/j.jcf.2017.08.013

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 31 Aug 2026.

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