Article

Proteostasis Landscapes of Cystic Fibrosis Variants Reveals Drug Response Vulnerability

2024-07-11

Abstract excerpt

<h4>ABSTRACT</h4> Cystic Fibrosis (CF) is a lethal genetic disorder caused by variants in CF transmembrane conductance regulator (CFTR). Many variants are treatable with correctors, which enhance the folding and trafficking of CFTR. However, approximately 3% of persons with CF harbor poorly responsive variants. Here, we used affinity purification mass spectrometry proteomics to profile the protein homeostasis (pr...

Topics

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

Identifiers and source

Literature Corpus work: 9d4a25ea-47c7-5e31-9bec-c930a2887165
DOI: 10.1101/2024.07.10.602964

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Proteostasis Landscapes of Cystic Fibrosis Variants Reveals Drug Response VulnerabilityDOI 10.1101/2024.07.10.602964Read

CFTR Folding: From Structure and Proteostasis to Cystic Fibrosis Personalized Medicine.Semantic proximity Read

Distinct proteostasis states drive pharmacologic chaperone susceptibility for Cystic Fibrosis Transmembrane Conductance Regulator misfolding mutantsSemantic proximity Read

Distinct proteostasis states drive pharmacologic chaperone susceptibility for cystic fibrosis transmembrane conductance regulator misfolding mutants.Semantic proximity Read

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

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

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

Small molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding statesSemantic proximity Read

Transcriptomic and Proteostasis Networks of CFTR and the Development of Small Molecule Modulators for the Treatment of Cystic Fibrosis Lung Disease.Semantic proximity Read

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

Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR.Semantic proximity Read

Comprehensive mapping of cystic fibrosis mutations to CFTR protein identifies mutation clusters and molecular docking predicts corrector binding site.Semantic 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