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机译:Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits
Univ Massachusetts, Sch Med, Dept Med, Worcester, MA 01655 USA;
Univ Massachusetts, Sch Med, Dept Biochem & Mol Pharmacol, Worcester, MA 01655 USA;
Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USAAlnylam Pharmaceut, Cambridge, MA 02142 USA;
gene delivery; gene silencing; Huntington's disease; neurodegenerative disease; RNAi; NEURONAL INTRANUCLEAR INCLUSIONS; MOUSE MODEL; RNA INTERFERENCE; IN-VIVO; MEDIATED DELIVERY; DISEASE; BRAIN; GENE; ABNORMALITIES; EXPRESSION;