Adeno-associated virus-mediated aspartoacylase gene transfer to the brain of knockout mouse for canavan disease

Molecular Therapy

Volumn 7, Issue 5 I, 2003, Pages 580-587

  Matalon, Reuben ^a   Surendran, Sankar ^a   Rady, Peter L ^a   Quast, Michael J ^a   Campbell, Gerald A ^b   Matalon, Kimberlee M ^a   Tyring, Stephen K ^a   Wei, Jingna ^a   Peden, Carmen S ^c   Ezell, Ed L ^a   Muzyczka, Nicholas ^c   Mandel, Ronald J ^c  

^a UNIVERSITY OF TEXAS MEDICAL BRANCH   (United States)

^b University of Texas Medical Branch School of Medicine   (United States)

^c UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

AAV GFP; Aspartoacylase; Canavan; Canavan mice; Gene transfer; rAAV hASPA

Indexed keywords

ASPARTOACYLASE; COMPLEMENTARY DNA; GREEN FLUORESCENT PROTEIN; N ACETYLASPARTIC ACID; PARVOVIRUS VECTOR; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; AUTOSOMAL RECESSIVE DISORDER; BRAIN DEGENERATION; BRAIN SPONGIOSIS; CANAVAN DISEASE; CONTROLLED STUDY; CORPUS STRIATUM; DRUG EFFECT; ENZYME ACTIVITY; ENZYME ASSAY; ENZYME DEFICIENCY; ENZYME REPLACEMENT; GENE CONSTRUCT; GENE TARGETING; GENE TRANSFER; GENETIC CODE; HISTOPATHOLOGY; HUMAN; IN VIVO STUDY; INJECTION SITE; KNOCKOUT MOUSE; LEUKODYSTROPHY; MOUSE; NEUROPATHOLOGY; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; PROTEIN EXPRESSION; REVIEW; RIBOSOME; THALAMUS; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; WESTERN BLOTTING;

ADENO-ASSOCIATED VIRUS; ANIMALIA; PARVOVIRUS;

EID: 0038529698     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1525-0016(03)00066-2     Document Type: Review

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