Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

Human Gene Therapy

Volumn 29, Issue 6, 2018, Pages 663-673

  Pfister, Edith L ^a   Dinardo, Natalie ^a   Mondo, Erica ^a   Borel, Florie ^a   Conroy, Faith ^a   Fraser, Cara ^b   Gernoux, Gwladys ^a   Han, Xin ^c   Hu, Danjing ^c   Johnson, Emily ^a,d   Kennington, Lori ^a   Liu, Pengpeng ^c   Reid, Suzanne J ^e   Sapp, Ellen ^f   Vodicka, Petr ^f,g   Kuchel, Tim ^b   Morton, A Jennifer ^h   Howland, David ⁱ   Moser, Richard ^a   Sena Esteves, Miguel ^a   Gao, Guangping ^a   Mueller, Christian ^a   Difiglia, Marian ^f   Aronin, Neil ^a   more..

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^b SOUTH AUSTRALIAN HEALTH AND MEDICAL RESEARCH INSTITUTE   (Australia)

^c SICHUAN UNIVERSITY   (China)

^d DARTMOUTH MEDICAL SCHOOL   (United States)

^e UNIVERSITY OF AUCKLAND   (New Zealand)

^f MASSACHUSETTS GENERAL HOSPITAL   (United States)

^g INSTITUTE OF ANIMAL PHYSIOLOGY AND GENETICS   (Czech Republic)

^h UNIVERSITY OF CAMBRIDGE   (United Kingdom)

ⁱ CHDI MANAGEMENT CHDI FOUNDATION   (United States)

Author keywords

AAV; Huntington's disease; large animal models; RNAi

Indexed keywords

ARTIFICIAL MICRORNA; HUNTINGTIN; MICRORNA; UNCLASSIFIED DRUG; ELECTROLYTE; HTT PROTEIN, HUMAN; MESSENGER RNA; MUTANT PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CAG REPEAT; CAUDATE NUCLEUS; CONTROLLED STUDY; CORPUS STRIATUM; DNA DETERMINATION; DROPLET DIGITAL POLYMERASE CHAIN REACTION; ENHANCER REGION; HEK293 CELL LINE; HUNTINGTON CHOREA; IMMUNOHISTOCHEMISTRY; INTRON; LEFT HEMISPHERE; LIQUORRHEA; NERVE CELL; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; POLYACRYLAMIDE GEL ELECTROPHORESIS; PROMOTER REGION; PROTEIN ANALYSIS; PUTAMEN; RIGHT HEMISPHERE; SINGLE DRUG DOSE; TRANSGENIC SHEEP; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ANIMAL; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE VECTOR; GENETICS; HUMAN; IMMUNOASSAY; INJECTION; KIDNEY; LIVER; METABOLISM; MICROGLIA; NEOSTRIATUM; PATHOLOGY; PATHOPHYSIOLOGY; SHEEP; TRANSGENIC ANIMAL; VIRUS GENOME;

ANIMALS; ANIMALS, GENETICALLY MODIFIED; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; ELECTROLYTES; GENETIC VECTORS; GENOME, VIRAL; HUMANS; HUNTINGTIN PROTEIN; HUNTINGTON DISEASE; IMMUNOASSAY; INJECTIONS; KIDNEY; LIVER; MICROGLIA; MICRORNAS; MUTANT PROTEINS; NEOSTRIATUM; NEURONS; RNA, MESSENGER; SHEEP;

EID: 85044582983     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2017.199     Document Type: Article

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