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Molecular Therapy

Nonallele-specific Silencing of Mutant and Wild-type Huntingtin Demonstrates Therapeutic Efficacy in Huntington's Disease Mice

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Molecular Therapy, 2009 Cited by 340 papers doi:10.1038/mt.2009.17

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HD mice showed significant reductions in wild-type and mutant htt, and demonstrated improved motor coordination and survival
Molecular Therapy · Feb 24, 2009
Huntington's disease (HD) is a fatal neurodegenerative disease caused by mutant huntingtin (htt) protein
Molecular Therapy · Feb 24, 2009
silencing mutant htt via RNA interference (RNAi) provides therapeutic benefit in HD mice
Molecular Therapy · Feb 24, 2009

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