Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease

Human Gene Therapy

Volumn 27, Issue 7, 2016, Pages 509-521

  Karumuthil Melethil, Subha ^a   Nagabhushan Kalburgi, Sahana ^a   Thompson, Patrick ^b   Tropak, Michael ^c   Kaytor, Michael D ^d   Keimel, John G ^d   Mark, Brian L ^e   Mahuran, Don ^c,f   Walia, Jagdeep S ^b   Gray, Steven J ^a  

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

^b QUEEN S UNIVERSITY   (Canada)

^c HOSPITAL FOR SICK CHILDREN   (Canada)

^d New Hope Research Foundation   (United States)

^e UNIVERSITY OF MANITOBA   (Canada)

^f UNIVERSITY OF TORONTO   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE ALPHA CHAIN; BETA N ACETYLHEXOSAMINIDASE ALPHA CHAIN MU; GANGLIOSIDE GM2; HOMODIMER; UNCLASSIFIED DRUG; BETA N ACETYLHEXOSAMINIDASE;

ADENO ASSOCIATED VIRUS; ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; FEMALE; GENE EXPRESSION; GENE REPLACEMENT THERAPY; INTRON; MOUSE; NERVE CELL; NEWBORN; NONHUMAN; PROMOTER REGION; PROTEIN PROTEIN INTERACTION; TAY SACHS DISEASE; VIRUS CAPSID; ADMINISTRATION AND DOSAGE; ANIMAL; C57BL MOUSE; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; LIVER; METABOLISM; MUTATION; TAY-SACHS DISEASE;

ANIMALS; ANIMALS, NEWBORN; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FEMALE; G(M2) GANGLIOSIDE; GENETIC THERAPY; GENETIC VECTORS; HEXOSAMINIDASES; LIVER; MICE; MICE, INBRED C57BL; MUTATION; TAY-SACHS DISEASE;

EID: 84987624678     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2016.013     Document Type: Article

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