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Article

Transformative therapies for rare CFTR missense alleles.

Current opinion in pharmacology - 1 Jun 2017

Oliver Kathryn E, Han Sangwoo T, Sorscher Eric J, Cutting Garry R

Abstract excerpt

With over 1900 variants reported in the cystic fibrosis transmembrane conductance regulator (CFTR), enhanced understanding of cystic fibrosis (CF) genotype-phenotype correlation represents an important and expanding area of research. The potentiator Ivacaftor has proven an effective treatment for a subset of individuals carrying missense variants, particularly those that impact CFTR gating. Therapeutic efforts...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 29032041
DOI: 10.1016/j.coph.2017.09.018
PMCID: PMC5723219

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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