The CFTR Corrector, VX-809 (Lumacaftor), Rescues ABCA4 Trafficking Mutants: a Potential Treatment for Stargardt Disease.
Cellular physiology and biochemistry : international journal of experimental cellular physiology, biochemistry, and pharmacology（2019）
Abstract
Our results provide a new mechanism for the rescue of ABCA4 trafficking mutants based on the restoration of Hsp27. Our results provide a pathway for the treatment of Stargardt disease.
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Key words
Chaperones,Common mutations,Correctors,Rescue,Stargardt disease
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