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Article

Rescue of NBD2 mutants N1303K and S1235R of CFTR by small-molecule correctors and transcomplementation.

PloS one - 1 Jan 2015

Rapino Daniele, Sabirzhanova Inna, Lopes-Pacheco Miquéias, Grover Rahul, Guggino William B, Cebotaru Liudmila

Abstract excerpt

Although, the most common Cystic Fibrosis mutation, ΔF508, in the cystic fibrosis transmembrane regulator. (CFTR), is located in nucleotide binding domain (NBD1), disease-causing mutations also occur in NBD2. To provide information on potential therapeutic strategies for mutations in NBD2, we studied, using a combination of biochemical approaches and newly created cell lines, two disease-causing NBD2 mutants,...

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Source

Open in PubMed Open full text

Identifiers

PMID: 25799511
DOI: 10.1371/journal.pone.0119796
PMCID: PMC4370480

Genes

CFTR

Provenance

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.

Related research

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Research trail→

Click a neighbor to travelStep 1 · 12 closest

Rescue of NBD2 mutants N1303K and S1235R of CFTR by small-molecule correctors and transcomplementation.PMID 25799511Read ↗

Rescue of CFTR NBD2 mutants N1303K and S1235R is influenced by the functioning of the autophagosome.Semantic proximity Read ↗

Correctors Rescue CFTR Mutations in Nucleotide-Binding Domain 1 (NBD1) by Modulating Proteostasis.Semantic proximity Read ↗

Combination of Correctors Rescue ΔF508-CFTR by Reducing Its Association with Hsp40 and Hsp27.Semantic proximity Read ↗

Preclinical Studies of a Rare CF-Causing Mutation in the Second Nucleotide Binding Domain (c.3700A>G) Show Robust Functional Rescue in Primary Nasal Cultures by Novel CFTR ModulatorsSemantic proximity Read ↗

Correctors of the Major Cystic Fibrosis Mutant Interact through Membrane-Spanning Domains.Semantic proximity Read ↗

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

The cystic fibrosis-causing mutation deltaF508 affects multiple steps in cystic fibrosis transmembrane conductance regulator biogenesis.Semantic proximity Read ↗

Functional rescue of c.3846G>A (W1282X) in patient-derived nasal cultures achieved by inhibition of nonsense mediated decay and protein modulators with complementary mechanisms of actionSemantic proximity Read ↗

Cystic fibrosis mutations lead to carboxyl-terminal fragments that highlight an early biogenesis step of the cystic fibrosis transmembrane conductance regulator.Semantic proximity Read ↗

Corrector-mediated rescue of misprocessed CFTR mutants can be reduced by the P-glycoprotein drug pump.Semantic proximity Read ↗

A new corrector rescues F508del-CFTR folding through stabilization of the TMD1-NBD1 linkerSemantic proximity Read ↗

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