AAV-based gene therapy prevents neuropathology and results in normal cognitive development in the hyperargininemic mouse

Gene Therapy

Volumn 20, Issue 8, 2013, Pages 785-796

  Lee, E K ^a   Hu, C ^a   Bhargava, R ^a   Ponnusamy, R ^a   Park, H ^a   Novicoff, S ^a   Rozengurt, N ^a   Marescau, B ^b   De Deyn, P ^b   Stout, D ^a   Schlichting, L ^c   Grody, W W ^a   Cederbaum, S D ^a,d,e   Lipshutz, G S ^a,d,e  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b UNIVERSITY OF ANTWERP   (Belgium)

^c UNIVERSITY OF COLORADO   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

AAV; brain; hyperargininemia

Indexed keywords

AMMONIA; ARGINASE 1;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BRAIN DEVELOPMENT; BRAIN DYSFUNCTION; COGNITIVE DEVELOPMENT; CONTROLLED STUDY; GENE EXPRESSION; GENE THERAPY; HISTOPATHOLOGY; HYPERARGININEMIA; MOUSE; NEUROPATHOLOGY; NEUROTOXICITY; NONHUMAN; PRIORITY JOURNAL; TRANSGENE;

ANIMALS; ARGINASE; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; HUMANS; HYPERAMMONEMIA; HYPERARGININEMIA; MICE; MICE, TRANSGENIC; NERVOUS SYSTEM DISEASES; NEURODEGENERATIVE DISEASES;

ADENO-ASSOCIATED VIRUS; MUS;

EID: 84881476043     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2012.99     Document Type: Article

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