Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's Disease

Human Gene Therapy

Volumn 25, Issue 5, 2014, Pages 461-474

  Stanek, Lisa M ^a   Sardi, Sergio P ^a   Mastis, Bryan ^a   Richards, Amy R ^a   Treleaven, Christopher M ^a   Taksir, Tatyana ^a   Misra, Kuma ^a   Cheng, Seng H ^a   Shihabuddin, Lamya S ^a  

^a SANOFI   (France)

Author keywords

[No Author keywords available]

Indexed keywords

HUNTINGTIN; PARVOVIRUS VECTOR; SMALL INTERFERING RNA; HTT PROTEIN, HUMAN; MESSENGER RNA; MICRORNA; MUTANT PROTEIN; NERVE PROTEIN;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BEHAVIOR DISORDER; CELL SELECTION; CLINICAL FEATURE; CORPUS STRIATUM; GENE SILENCING; HUNTINGTON CHOREA; IN VITRO STUDY; MOUSE; NEUROTOXICITY; NONHUMAN; PROTEIN EXPRESSION; RNA INTERFERENCE; RNA TRANSCRIPTION; ANIMAL; ANIMAL BEHAVIOR; DEPENDOPARVOVIRUS; DISEASE MODEL; GENETIC TRANSDUCTION; GENETICS; HEK293 CELL LINE; HUMAN; METABOLISM; NEOSTRIATUM; PATHOLOGY; TRANSGENIC MOUSE;

ANIMALS; BEHAVIOR, ANIMAL; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; HEK293 CELLS; HUMANS; HUNTINGTON DISEASE; MICE; MICE, TRANSGENIC; MICRORNAS; MUTANT PROTEINS; NEOSTRIATUM; NERVE TISSUE PROTEINS; RNA INTERFERENCE; RNA, MESSENGER; TRANSDUCTION, GENETIC;

EID: 84901195245     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2013.200     Document Type: Article

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