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ML-SA5 is a potent TRPML cation channel agonist that activates whole-endolysosomal TRPML1 (ML1) currents in DMD myoblasts at a higher potency than ML-SA1 (EC50 = 285 nM vs 10 μM). ML-SA5 ameliorates muscular dystrophy in mdx, but not ML1-knockout, mice in vivo (2 or 5 mg/kg/day i.p.) by facilitating sarcolemma repair, thereby reducing skeletal and cardiac muscle damage with no apparent toxicity to the animals.
Potent TRPML1 cation channel agonist that ameliorates muscular dystrophy in mdx, but not ML1-KO, mice and with a higher potency than ML-SA1.
储存分类代码
11 - Combustible Solids
WGK
WGK 3
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Not applicable
闪点(°C)
Not applicable
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