Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

Human Gene Therapy

Volumn 13, Issue 11, 2002, Pages 1391-1412

  Janson, Christopher ^a   McPhee, Scott ^c   Bilaniuk, Larissa ^b   Haselgrove, John ^c   Testaiuti, Mark ^a   Freese, Andrew ^c   Wang, Dah Jyuu ^c   Shera, David ^c   Hurh, Peter ^c   Rupin, Joan ^c   Saslow, Elizabeth ^c   Goldfarb, Olga ^c   Goldberg, Michael ^c   Larijani, Ghassem ^c   Sharrar, William ^c   Liouterman, Larisa ^c   Camp, Angelique ^c   Kolodny, Edwin ^c   Samulski, Jude ^c   Leone, Paola ^a  

^a RUTGERS ROBERT WOOD JOHNSON MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c UNIVERSITY OF NORTH CAROLINA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMIDASE; ASPARTOACYLASE; N ACETYLASPARTIC ACID;

ADENO ASSOCIATED VIRUS; ARTICLE; BRAIN; CANAVAN DISEASE; CLINICAL PROTOCOL; CLINICAL TRIAL; COHORT ANALYSIS; ENZYMOLOGY; FEMALE; FOLLOW UP; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HUMAN; INFANT; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; MALE; METABOLISM; METHODOLOGY; NUCLEAR MAGNETIC RESONANCE IMAGING; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; PATHOLOGY; PATHOPHYSIOLOGY; PATIENT SELECTION; PRESCHOOL CHILD; ASPA GENE; AUTOSOMAL RECESSIVE DISORDER; BIOCHEMISTRY; CENTRAL NERVOUS SYSTEM; DRUG SAFETY; FATALITY; GENETIC CODE; LEUKODYSTROPHY; MYELINATION; NERVE DEGENERATION; RADIOLOGY; SIDE EFFECT; VIRUS GENE; VIRUS GENOME; VIRUS RECOMBINANT;

AMIDOHYDROLASES; BRAIN; CANAVAN DISEASE; CHILD, PRESCHOOL; CLINICAL PROTOCOLS; CLINICAL TRIALS; COHORT STUDIES; DEPENDOVIRUS; FEMALE; FOLLOW-UP STUDIES; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; INFANT; INJECTIONS, INTRAVENTRICULAR; MAGNETIC RESONANCE IMAGING; MAGNETIC RESONANCE SPECTROSCOPY; MALE; PATIENT SELECTION;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 2;

EID: 0037143294     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/104303402760128612     Document Type: Article

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