Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo

Molecular Therapy Methods and Clinical Development

Volumn 3, Issue , 2016, Pages 15057-

  Tropak, Michael B ^a   Yonekawa, Sayuri ^a   Karumuthil Melethil, Subha ^b   Thompson, Patrick ^c   Wakarchuk, Warren ^d   Gray, Steven J ^b   Walia, Jagdeep S ^c   Mark, Brian L ^e   Mahuran, Don ^a,f  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

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

^c QUEEN S UNIVERSITY   (Canada)

^d RYERSON UNIVERSITY   (Canada)

^e UNIVERSITY OF MANITOBA   (Canada)

^f UNIVERSITY OF TORONTO   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE; BETA N ACETYLHEXOSAMINIDASE A; BETA N ACETYLHEXOSAMINIDASE B; GANGLIOSIDE GM2; GANGLIOSIDE GM2 ACTIVATOR PROTEIN; HOMODIMER; ISOENZYME; ADENO ASSOCIATED VIRUS VECTOR; BETA N ACETYLHEXOSAMINIDASE M; BETA N ACETYLHEXOSAMINIDASE S; UNCLASSIFIED DRUG;

ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; ENZYME REPLACEMENT; EXPERIMENTAL MODEL; FEMALE; GENE; GENE EDITING; GENE MUTATION; GENE REPLACEMENT THERAPY; GENE TRANSFER; HUMAN; HUMAN CELL; HYBRID; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NONHUMAN; PRIORITY JOURNAL; SANDHOFF DISEASE; TAY SACHS DISEASE; ANIMAL TISSUE; CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT; CONTROLLED STUDY; EMBRYO; ENZYME ACTIVE SITE; GENE CONSTRUCT; GENE DISRUPTION; GENE EXPRESSION; LIPID HYDROLYSIS; PROTEIN PROTEIN INTERACTION; PURIFICATION; VIRAL GENE DELIVERY SYSTEM;

EID: 84987595963     PISSN: None     EISSN: 23290501     Source Type: Journal    
DOI: 10.1038/mtm.2015.57     Document Type: Article

References (52)

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