F508 Corrector technologies and mechanistic assays available through the CFC
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چکیده
Overview: This document describes capabilities available through the CFTR Folding Consortium (CFC) and other groups pursuing F508 corrector screens and drug characterization. Included are specific areas of CFC investigator expertise, brief summaries of selected assays relevant to corrector mechanism of action, and new CFC protocols suitable for compound library screening. Academic and commercial laboratories interested in F508del correction are welcome to contact individual CFC members regarding availability of assays, establishment of collaborations, etc.
منابع مشابه
Requirements for Efficient Correction of ΔF508 CFTR Revealed by Analyses of Evolved Sequences
Misfolding of ΔF508 cystic fibrosis (CF) transmembrane conductance regulator (CFTR) underlies pathology in most CF patients. F508 resides in the first nucleotide-binding domain (NBD1) of CFTR near a predicted interface with the fourth intracellular loop (ICL4). Efforts to identify small molecules that restore function by correcting the folding defect have revealed an apparent efficacy ceiling. ...
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