Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease

Proceedings of the National Academy of Sciences of the United States of America

Volumn 96, Issue 5, 1999, Pages 2296-2300

  Daly, Thomas M ^a   Vogler, Carole ^b   Levy, Beth ^b   Haskins, Mark E ^c   Sands, Mark S ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

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

^c UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Adeno associated virus; Animal models; Gene therapy; Metabolic disease; Mucopolysaccharidosis type VII

Indexed keywords

BETA GLUCURONIDASE; MANNOSE 6 PHOSPHATE;

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CENTRAL NERVOUS SYSTEM; GENE CASSETTE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; LYSOSOME STORAGE DISEASE; MICROGLIA; MUCOPOLYSACCHARIDOSIS; NEWBORN; NONHUMAN; PATHOLOGY; PRIORITY JOURNAL; TISSUE DIFFERENTIATION;

ANIMALS; ANIMALS, NEWBORN; DEPENDOVIRUS; DNA, COMPLEMENTARY; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GLUCURONIDASE; GLYCOSAMINOGLYCANS; HUMANS; LIVER; LYSOSOMAL STORAGE DISEASES; LYSOSOMES; MICE; MICE, MUTANT STRAINS; MUCOPOLYSACCHARIDOSIS VII; POLYMERASE CHAIN REACTION; RECOMBINANT PROTEINS;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; ANIMALIA; MURINAE;

EID: 0033515007     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.96.5.2296     Document Type: Article

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