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Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.
Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients.
Linking SNPs to CAG repeat length in Huntington's disease patients.
Huntington's disease: silencing a brutal killer.
Increased Steady-State Mutant Huntingtin mRNA in Huntington's Disease Brain.
Startups on the menu: Alnylam.