Gene transfer corrects acute GM2 gangliosidosispotential therapeutic contribution of perivascular enzyme flow

Molecular Therapy

Volumn 20, Issue 8, 2012, Pages 1489-1500

  Begoacachon Gonzalez M ^a   Wang, Susan Z ^a   McNair, Rosamund ^a   Bradley, Josephine ^a   Lunn, David ^b   Ziegler, Robin ^c   Cheng, Seng H ^c   Cox, Timothy M ^a  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^b UNIVERSITY OF CAMBRIDGE   (United Kingdom)

^c GENZYME CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE; BETA N ACETYLHEXOSAMINIDASE ALPHA CHAIN; BETA N ACETYLHEXOSAMINIDASE BETA CHAIN; BETA N ACETYLHEXOSAMINIDASE S; CD68 ANTIGEN; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ATAXIA; BRAIN DISEASE; CONTROLLED STUDY; CORPUS STRIATUM; DRUG DISTRIBUTION; ENZYME ACTIVITY; ENZYME SYNTHESIS; ENZYME THERAPY; FEMALE; GENE TRANSFER; GM2 GANGLIOSIDOSIS; HISTOLOGY; ION EXCHANGE CHROMATOGRAPHY; MALE; MOUSE; NERVE DEGENERATION; NERVOUS SYSTEM INFLAMMATION; NONHUMAN; PROTEIN EXPRESSION; SANDHOFF DISEASE; SPASTICITY; SPINAL CORD DISEASE; SURVIVAL TIME; TREATMENT OUTCOME; TREMOR; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; WASTING SYNDROME;

MUS;

EID: 84864540429     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2012.44     Document Type: Article

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