AAV-Mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy

Molecular Therapy

Volumn 11, Issue 5, 2005, Pages 734-744

  Rafi, Mohammad A ^a   Zhi Rao, Han ^a   Passini, Marco A ^b,d   Curtis, Mark ^a   Vanier, Marie T ^c   Zaka, Mariam ^a   Luzi, Paola ^a   Wolfe, John H ^b   Wenger, David A ^a  

^a THOMAS JEFFERSON UNIVERSITY   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c CENTRE HOSPITALIER LYON SUD   (France)

^d GENZYME CORPORATION   (United States)

Author keywords

Adeno associated virus; Brain; Gene therapy; Gene transfer techniques; Krabbe disease; Leukodystrophy; Lysosomal storage disease; myelination; Psychosine

Indexed keywords

GALACTOSYLCERAMIDASE; PARVOVIRUS VECTOR; RECOMBINANT DNA;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DISEASE MODEL; ENZYME ACTIVITY; FATALITY; GLOBOID CELL LEUKODYSTROPHY; HUMAN CYTOMEGALOVIRUS; MOUSE; MOUSE STRAIN; MYELINATION; NEWBORN; NONHUMAN; ONSET AGE; PROTEIN EXPRESSION; SEROTYPE; STRAIN DIFFERENCE; SURVIVAL; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS CAPSID;

ANIMALS; BODY WEIGHT; BRAIN; CELLS, CULTURED; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; GALACTOSYLCERAMIDASE; GENETIC VECTORS; HUMANS; IMMUNOHISTOCHEMISTRY; LEUKODYSTROPHY, GLOBOID CELL; MICE; PSYCHOSINE; SURVIVAL RATE;

ADENO-ASSOCIATED VIRUS; ANIMALIA; HUMAN HERPESVIRUS 5; MURINAE;

EID: 17644374493     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.12.020     Document Type: Article

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