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Genomics
Comparison of CFTR Theratype Selectivity and Off-Target Interactions of VX-121 and VX-445
PRJNA1471632 | ENA
UnknownOrkambi® and amplifier co-therapy improves function from a rare CFTR mutation in gene-edited cells and patient tissue.
Not available
S-EPMC5582412 | biostudies-literature
UnknownCirculating Truncated Alpha-1 Antitrypsin Glycoprotein in Patient Plasma Retains Anti-Inflammatory Capacity.
Not available
S-EPMC6452030 | biostudies-literature
UnknownBenchmarking AlphaMissense Pathogenicity Predictions Against Cystic Fibrosis Variants.
Not available
S-EPMC10592606 | biostudies-literature
UnknownBenchmarking AlphaMissense pathogenicity predictions against cystic fibrosis variants.
Not available
S-EPMC10810519 | biostudies-literature
ProteomicsDistinct proteostasis states drive pharmacologic chaperone susceptibility for Cystic Fibrosis Transmembrane Conductance Regulator misfolding mutants
Pharmacological chaperones represent a class of therapeutic compounds for treating protein misfolding diseases. One of the most prominent examples is the FDA-approved pharmacological chaperone lumacaftor (VX-809), which has transformed cystic fibrosis (CF) therapy. CF is a fatal disease caused by mu...
ORGANISM(S): Homo sapiens (Human) 
2022-05-04 | PXD032836 | Pride
Cystic fibrosis Protein quality control Corrector Interactomics Affinity-purification mass spectrometry (ap-ms) Theratype Tandem mass tags 
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