Sustained correction of OTC deficiency in spf ash mice using optimized self-complementary AAV2/8 vectors

Gene Therapy

Volumn 19, Issue 4, 2012, Pages 404-410

  Wang, L ^a   Wang, H ^a,c   Morizono, H ^b   Bell, P ^a   Jones, D ^b   Lin, J ^a   McMenamin, D ^a   Yu, H ^a   Batshaw, M L ^b   Wilson, J M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b CHILDREN S RESEARCH INSTITUTE   (United States)

^c SUN YAT SEN UNIVERSITY   (China)

Author keywords

adeno associated viruses; liver gene therapy; OTC deficiency; self complementary

Indexed keywords

ADENOVIRUS VECTOR; COMPLEMENTARY DNA; ORNITHINE CARBAMOYLTRANSFERASE; OROTIC ACID; THYROXINE BINDING GLOBULIN;

ACIDURIA; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; ENZYME ACTIVITY; GENE CONSTRUCT; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; IMMUNOHISTOCHEMISTRY; MOUSE; NONHUMAN; ORNITHINE TRANSCARBAMYLASE DEFICIENCY; PRIORITY JOURNAL; PROMOTER REGION; SEQUENCE ANALYSIS; SINGLE DRUG DOSE; THERAPY EFFECT; TRANSGENE;

ANIMALS; BASE SEQUENCE; CONSERVED SEQUENCE; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE THERAPY; GENETIC VECTORS; INJECTIONS, INTRAVENOUS; MICE; ORNITHINE CARBAMOYLTRANSFERASE; ORNITHINE CARBAMOYLTRANSFERASE DEFICIENCY DISEASE; OROTIC ACID; SPECIFIC PATHOGEN-FREE ORGANISMS; THYROXINE-BINDING GLOBULIN;

ADENO-ASSOCIATED VIRUS; MURINAE; MUS;

EID: 84859596214     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2011.111     Document Type: Article

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