生物活性 | |||
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描述 | Mutant huntingtin protein (mHTT) contains an expanded polyglutamine tract and causes Huntington’s disease (HD). Microtubule-associated proteins 1A/1B light chain 3B (LC3) is a protein associated with autophagy substrate selection and autophagosome biogenesis. mHTT can be degraded by autophagy, during which protein substrates are incorporated into autophagosomes associated with LC3. LC3-mHTT-IN-AN1 is a mHTT-LC3 linker compound that interacts with both mHTT and LC3, but not with wild-type HTT. LC3-mHTT-IN-AN1 at concentrations of 10 – 300nM reduced the level of mHTT in cultured HD mouse neurons in an allele-selective manner. LC3-mHTT-IN-AN1 also reduced the level of mutant but not wild-type ATXN3 in primary fibroblasts from patients with spinocerebellar ataxia type 3. Treatment with LC3-mHTT-IN-AN1 (2 μl at 25 μM per day) for 10 days led to a significant lowering of mHTT in the cortices of HD mice[1]. |
实验方案 | |||
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1mg | 5mg | 10mg | |
1 mM 5 mM 10 mM |
2.43mL 0.49mL 0.24mL |
12.16mL 2.43mL 1.22mL |
24.33mL 4.87mL 2.43mL |
参考文献 |
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