AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice

Molecular Genetics and Metabolism

Volumn 120, Issue 4, 2017, Pages 299-305

  Wang, Lili ^a   Bell, Peter ^b   Morizono, Hiroki ^c   He, Zhenning ^b   Pumbo, Elena ^c   Yu, Hongwei ^b   White, John ^b   Batshaw, Mark L ^c   Wilson, James M ^b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c CHILDREN S RESEARCH INSTITUTE   (United States)

Author keywords

AAV; Gene therapy; Liver fibrosis; OTC deficiency

Indexed keywords

OROTIC ACID; ORNITHINE CARBAMOYLTRANSFERASE;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; FEMALE; GENE MUTATION; GENOME; HISTOPATHOLOGY; KNOCKOUT MOUSE; LIVER FIBROSIS; MOUSE; NONHUMAN; ORNITHINE TRANSCARBAMYLASE DEFICIENCY; PRIORITY JOURNAL; UREA CYCLE; VIRAL GENE THERAPY; ADMINISTRATION AND DOSAGE; AGING; ANIMAL; COMPLICATION; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; LIVER CIRRHOSIS; MALE; ORNITHINE CARBAMOYLTRANSFERASE DEFICIENCY DISEASE; TREATMENT OUTCOME;

AGING; ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FEMALE; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LIVER CIRRHOSIS; MALE; MICE; MICE, KNOCKOUT; ORNITHINE CARBAMOYLTRANSFERASE; ORNITHINE CARBAMOYLTRANSFERASE DEFICIENCY DISEASE; TREATMENT OUTCOME;

EID: 85014507503     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2017.02.011     Document Type: Article

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