Article

Profiling the CFTR Variant Selectivity and Off-Target Interactions of VX-121

2026-06-04

Abstract excerpt

More than 1,200 variants of the cystic fibrosis transmembrane conductance regulator gene ( CFTR ) are associated with cystic fibrosis (CF), an autosomal recessive pulmonary disease affecting over 100,000 people. Most people with CF bear a common CFTR variant (F508del) that can be treated with therapeutics containing “correctors” that suppress the misfolding of the CFTR chloride channel. However, the pharmacologi...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 418dce7c-70b8-57da-ba28-c846b190f454
DOI: 10.64898/2026.06.01.729306

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Profiling the CFTR Variant Selectivity and Off-Target Interactions of VX-121DOI 10.64898/2026.06.01.729306Read

Elucidation of Global Trends in the Effects of VX-661 and VX-445 on the Expression of Clinical CFTR VariantsSemantic proximity Read

General trends in the effects of VX-661 and VX-445 on the plasma membrane expression of clinical CFTR variants.Semantic proximity Read

Ivacaftor: the first therapy acting on the primary cause of cystic fibrosis.Semantic proximity Read

Mutation-specific downregulation of CFTR2 variants by gating potentiators.Semantic proximity Read

Ivacaftor treatment of cystic fibrosis patients with the G551D mutation: a review of the evidence.Semantic proximity Read

General Trends in the Calnexin-Dependent Expression and Pharmacological Rescue of Clinical CFTR VariantsSemantic proximity Read

General trends in the calnexin-dependent expression and pharmacological rescue of clinical CFTR variants.Semantic proximity Read

Elexacaftor/VX-445-mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamicsSemantic proximity Read

PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR.Semantic proximity Read

Rescue of Rare CFTR Trafficking Mutants Highlights a Structural Location-Dependent Pattern for Correction.Semantic proximity Read

Mechanism-based corrector combination restores ΔF508-CFTR folding and function.Semantic proximity Read

Fluorescence assay for simultaneous quantification of CFTR ion-channel function and plasma membrane proximitySemantic proximity Read