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Article

Comprehensive Assessment of CFTR Modulators' Therapeutic Efficiency for N1303K Variant.

International journal of molecular sciences - 27 Feb 2024

Efremova Anna, Kashirskaya Nataliya, Krasovskiy Stanislav, Melyanovskaya Yuliya, Krasnova Maria, Mokrousova Diana, Bulatenko Nataliya, Kondratyeva Elena, Makhnach Oleg, Bukharova Tatiana, Zinchenko Rena, Kutsev Sergey, Goldshtein Dmitry

Abstract excerpt

p.Asn1303Lys (N1303K) is a common missense variant of the CFTR gene, causing cystic fibrosis (CF). In this study, we initially evaluated the influence of CFTR modulators on the restoration of N1303K-CFTR function using intestinal organoids derived from four CF patients expressing the N1303K variant. The forskolin-induced swelling assay in organoids offered valuable insights about the beneficial effects of VX-770...

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Identifiers

PMID: 38474016
DOI: 10.3390/ijms25052770
PMCID: PMC10931982

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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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