Article
Corrector VX-809 stabilizes the first transmembrane domain of CFTR.
Biochemical pharmacology - 1 Sept 2013
Loo Tip W, Bartlett M Claire, Clarke David M
Abstract excerpt
Processing mutations that inhibit folding and trafficking of CFTR are the main cause of cystic fibrosis (CF). A potential CF therapy would be to repair CFTR processing mutants. It has been demonstrated that processing mutants of P-glycoprotein (P-gp), CFTR's sister protein, can be efficiently repaired by a drug-rescue mechanism. Many arginine suppressors that mimic drug-rescue have been identified in the P-gp...
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