Pathogenesis of growth failure and partial reversal with gene therapy in murine and canine Glycogen Storage Disease type Ia

Molecular Genetics and Metabolism

Volumn 109, Issue 2, 2013, Pages 161-170

  Brooks, Elizabeth Drake ^a   Little, Dianne ^a   Arumugam, Ramamani ^a   Sun, Baodong ^a   Curtis, Sarah ^a   DeMaster, Amanda ^a   Maranzano, Michael ^a   Jackson, Mark W ^b   Kishnani, Priya ^a   Freemark, Michael S ^a   Koeberl, Dwight D ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b UNIVERSITY OF GLASGOW   (United Kingdom)

Author keywords

Adeno associated virus; Gene therapy; Glucose 6 phosphatase; Glycogen Storage Disease Ia; Growth failure; IGF 1

Indexed keywords

ADENOVIRUS VECTOR; GLUCOSE 6 PHOSPHATASE; GROWTH HORMONE; SOMATOMEDIN C; STAT5 PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BODY WEIGHT; BONE DYSPLASIA; BONE MALFORMATION; BONE SHORTENING; CONTROLLED STUDY; DOG; GENE THERAPY; GLYCOGEN STORAGE DISEASE TYPE 1; GLYCOGEN STORAGE DISEASE TYPE 1A; GROWTH DISORDER; LEG MALFORMATION; MOUSE; NONHUMAN; PATHOGENESIS; PRIORITY JOURNAL;

ANIMALS; BONE AND BONES; DOGS; FEMALE; GENETIC THERAPY; GLUCOSE-6-PHOSPHATASE; GLYCOGEN; GLYCOGEN STORAGE DISEASE TYPE I; GROWTH HORMONE; INSULIN-LIKE GROWTH FACTOR I; LIPID METABOLISM; LIVER; MALE; MICE; MICE, KNOCKOUT; OSTEOGENESIS;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS - 8; CANIS FAMILIARIS; MURINAE; MUS;

EID: 84878489281     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2013.03.018     Document Type: Article

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