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Article

Genomically-guided therapies: A new era for cystic fibrosis.

Archives de pediatrie : organe officiel de la Societe francaise de pediatrie - 1 Feb 2020

Fajac I, Girodon E

Abstract excerpt

Since the cloning of the CFTR gene 30 years ago, research aiming at understanding how CFTR mutations translate to abnormal synthesis or function of the CFTR protein has opened the way to genomically-guided therapy to improve CFTR function. A CFTR potentiator to enhance CFTR channel function has been approved in 2012 for specific and quite rare mutations. Subsequently, combinations of a corrector to increase CFTR...

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PMID: 32172937
DOI: 10.1016/S0929-693X(20)30050-6

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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