Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

Gene Therapy

Volumn 8, Issue 17, 2001, Pages 1291-1298

  Daly, T M ^a   Ohlemiller, K K ^b   Roberts, M S ^a   Vogler, C A ^c   Sands, M S ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b CENTRAL INSTITUTE FOR THE DEAF   (United States)

^c SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Animal models; Gene therapy; Lysosomal storage disease; Metabolic disease

Indexed keywords

BETA GLUCURONIDASE; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BODY WEIGHT; BONE; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; DRUG DISTRIBUTION; FEMALE; GENE TRANSFER; HEARING; INBORN ERROR OF METABOLISM; LIFE; LYSOSOME STORAGE DISEASE; MALE; MOUSE; MOUSE MUTANT; MUCOPOLYSACCHARIDOSIS TYPE 7; NEWBORN; NEWBORN DISEASE; NEWBORN PERIOD; NONHUMAN; PRIORITY JOURNAL; RETINA; SIBLING; SURVIVAL; SYMPTOMATOLOGY; VIRUS RECOMBINANT;

ANIMALS; ANIMALS, NEWBORN; DEAFNESS; ELECTRORETINOGRAPHY; EVOKED POTENTIALS, AUDITORY, BRAIN STEM; GENE EXPRESSION; GENE THERAPY; GLUCURONIDASE; MICE; MICE, MUTANT STRAINS; MODELS, ANIMAL; MUCOPOLYSACCHARIDOSIS VII; RETINA; SURVIVAL RATE; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; ANIMALIA; DNA VIRUSES; MURINAE;

EID: 0034824239     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3301420     Document Type: Article

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