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Combating Cystic Fibrosis: In Search for CF Transmembrane Conductance Regulator (CFTR) Modulators
Author(s) -
Noy Efrat,
Senderowitz Hanoch
Publication year - 2011
Publication title -
chemmedchem
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.817
H-Index - 100
eISSN - 1860-7187
pISSN - 1860-7179
DOI - 10.1002/cmdc.201000488
Subject(s) - cystic fibrosis transmembrane conductance regulator , cystic fibrosis , regulator , gating , transmembrane protein , potentiator , microbiology and biotechnology , chemistry , computational biology , medicine , biology , neuroscience , pharmacology , biochemistry , receptor , gene
Open sesame! Compounds that correct the folding, trafficking and gating defects in cystic fibrosis transmembrane conductance regulator (CFTR), the main protein implicated in cystic fibrosis (CF), are of great interest as potential therapeutic agents for this lethal disease. The development of CFTR modulators, highlighting the main challenges in the field and advocating the application of rational rather than brute‐force methods, is the focus of this Minireview.