Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice

Human Gene Therapy

Volumn 27, Issue 7, 2016, Pages 497-508

  Osmon, Karlaina J L ^a   Woodley, Evan ^b   Thompson, Patrick ^b   Ong, Katalina ^b   Karumuthil Melethil, Subha ^c   Keimel, John G ^d   Mark, Brian L ^e   Mahuran, Don ^f,g   Gray, Steven J ^c   Walia, Jagdeep S ^a,b  

^a QUEEN S UNIVERSITY   (Canada)

^b QUEEN S UNIVERSITY   (Canada)

^c UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^d New Hope Research Foundation   (United States)

^e UNIVERSITY OF MANITOBA   (Canada)

^f HOSPITAL FOR SICK CHILDREN   (Canada)

^g UNIVERSITY OF TORONTO   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE; BETA N ACETYLHEXOSAMINIDASE ALPHA CHAIN; GANGLIOSIDE GM2; GANGLIOSIDE GM2 ACTIVATOR PROTEIN; HEXOSAMINIDASE MU SUBUNIT; HOMODIMER; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BEHAVIOR; BETA CHAIN; BIOCHEMICAL ANALYSIS; BRAIN REGION; COHORT ANALYSIS; CONTROLLED STUDY; ENZYME ANALYSIS; GENE FUNCTION; GENETIC VARIABILITY; GM2 GANGLIOSIDOSIS; MOUSE; NEWBORN; NONHUMAN; PROTEIN PROTEIN INTERACTION; SANDHOFF DISEASE; SINGLE DRUG DOSE; SPINAL CORD; SURVIVAL ANALYSIS; TREATMENT DURATION; VIRAL GENE DELIVERY SYSTEM; ADMINISTRATION AND DOSAGE; ANIMAL; ANIMAL BEHAVIOR; DEPENDOPARVOVIRUS; DISEASE MODEL; ENZYMOLOGY; GENE THERAPY; GENE VECTOR; GENETICS; MUTATION; PHENOTYPE;

ANIMALS; ANIMALS, NEWBORN; BEHAVIOR, ANIMAL; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; HEXOSAMINIDASES; MICE; MUTATION; PHENOTYPE; SANDHOFF DISEASE;

EID: 84987680747     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2016.015     Document Type: Article

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