Selective inhibitors of death in mutant huntingtin cells |
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Authors: | Varma Hemant Voisine Cindy DeMarco C Todd Cattaneo Elena Lo Donald C Hart Anne C Stockwell Brent R |
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Institution: | Department of Biological Sciences, Columbia University, Fairchild Center, MC 2406, 1212 Amsterdam Avenue, New York, New York 10027, USA. |
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Abstract: | 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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