Neurological correction of lysosomal storage in a mucopolysaccharidosis IIIB mouse model by adeno-associated virus-mediated gene delivery

Molecular Therapy

Volumn 5, Issue 1, 2002, Pages 42-49

  Fu, Haiyan ^a   Samulski, Richard J ^a   McCown, Thomas J ^a   Picornell, Yoana J ^a   Fletcher, David ^a   Muenzer, Joseph ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Lysosomal storage; Mucopolysaccharidosis IIIB; N acetylglucosaminidase

Indexed keywords

ALPHA N ACETYLGLUCOSAMINIDASE; COMPLEMENTARY DNA; GLYCOSAMINOGLYCAN; NEURON SPECIFIC ENOLASE; RECOMBINANT ENZYME; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BIOACCUMULATION; BRAIN CELL; BRAIN NERVE CELL; CELL CULTURE; CONTROLLED STUDY; CYTOMEGALOVIRUS; FIBROBLAST; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSDUCTION; HISTOLOGY; HUMAN; HUMAN CELL; IN VITRO STUDY; LYSOSOME STORAGE DISEASE; MICROINJECTION; MOUSE; NEUROLOGIC DISEASE; NONHUMAN; PROMOTER REGION; SANFILIPPO SYNDROME; TARGET CELL; VIRAL GENE DELIVERY SYSTEM; VIRUS LOAD; VIRUS RECOMBINANT;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CUCUMBER MOSAIC VIRUS; CYTOMEGALOVIRUS;

EID: 0036154761     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2001.0514     Document Type: Article

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