期刊
NATURE CHEMICAL BIOLOGY
卷 3, 期 2, 页码 99-100出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nchembio852
关键词
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资金
- Telethon [GGP06250] Funding Source: Medline
Huntington disease (HD) is an inherited neurodegenerative disorder with unclear pathophysiology. We developed a high-throughput assay in a neuronal cell culture model of HD, screened 43,685 compounds and identified 29 novel selective inhibitors of cell death in mutant huntingtin - expressing cells. Four compounds were active in diverse HD models, which suggests a role for cell death in HD; these compounds are mechanistic probes and potential drug leads for HD.
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