Article
Increasing the Endoplasmic Reticulum Pool of the F508del Allele of the Cystic Fibrosis Transmembrane Conductance Regulator Leads to Greater Folding Correction by Small Molecule Therapeutics.
PloS one - 1 Jan 2016
Chung W Joon, Goeckeler-Fried Jennifer L, Havasi Viktoria, Chiang Annette, Rowe Steven M, Plyler Zackery E, Hong Jeong S, Mazur Marina, Piazza Gary A, Keeton Adam B, White E Lucile, Rasmussen Lynn, Weissman Allan M, Denny R Aldrin, Brodsky Jeffrey L, Sorscher Eric J
Abstract excerpt
Small molecules that correct the folding defects and enhance surface localization of the F508del mutation in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) comprise an important therapeutic strategy for cystic fibrosis lung disease. However, compounds that rescue the F508del mutant protein to wild type (WT) levels have not been identified. In this report, we consider obstacles to obtaining robust...
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