TRPML 1,2 and 3 channel activator.1 Does not activate TRPM2, TRPV2, TRPV3, TRPC6 or TRPA1 channels.2 ML-SA1 robustly activates whole cell TRPML1-4A and whole-endolysosome TRPML1 and is comparable (10 µM) to the effect of the endogenous TRPML agonist, PI(3,5)P2 (1 µM).2 Thus it is a useful chemical tool for studying the functions of TRPMLs. It induces TRPML-mediated Ca2+ release from lysosomes which corrects trafficking defects and reduces cholesterol accumulation in Niemann-Pick type C macrophages.2,3 Reduces intralysosomal Ca2+ level rescuing abnormal lysosomal storage in FIG4-deficient cells.4
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