Article
F508del-cystic fibrosis transmembrane regulator correctors for treatment of cystic fibrosis: a patent review.
Expert opinion on therapeutic patents - 1 Jan 2015
Yang Hong, Ma Tonghui
Abstract excerpt
INTRODUCTION: Cystic fibrosis (CF) is an autosomal recessive genetic disease caused by malfunction of CF transmembrane regulator (CFTR). The deletion of a phenylalanine at residue 508 (F508del) is the most common mutation that causes cellular processing, chloride channel gating and protein stability defects in CFTR. Pharmacological modulators of F508del-CFTR, aimed at correcting the cellular processing defect...
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