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Article

Splicing mutations in the CFTR gene as therapeutic targets.

Gene therapy - 1 Aug 2022

Deletang Karine, Taulan-Cadars Magali

Abstract excerpt

The marketing approval, about ten years ago, of the first disease modulator for patients with cystic fibrosis harboring specific CFTR genotypes (~5% of all patients) brought new hope for their treatment. To date, several therapeutic strategies have been approved and the number of CFTR mutations targeted by therapeutic agents is increasing. Although these drugs do not reverse the existing disease, they help to...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 35650428
DOI: 10.1038/s41434-022-00347-0
PMCID: PMC9385490

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

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