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Protein suppressor gene offers Huntington's disease hope
Researchers report that inserting an artificial protein gene into cells with the Huntington's mutation lowers the lethal Htt protein levels and reduces nerve cell death in fruit flies. The approach may guide drug therapies to disrupt Htt buildup, with gene therapy as one possible route though delivery challenges exist. The work, led by Thompson and Marsh with MIT and UCI colleagues, is funded by multiple foundations and NIH.
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Central Nervous System
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