Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome

Molecular Genetics and Metabolism

Volumn 105, Issue 2, 2012, Pages 203-211

  Wang, Lili ^a   Morizono, Hiroki ^b   Lin, Jianping ^a   Bell, Peter ^a   Jones, David ^b   McMenamin, Deirdre ^a   Yu, Hongwei ^a   Batshaw, Mark L ^b   Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b CHILDREN S RESEARCH INSTITUTE   (United States)

Author keywords

Adeno associated viruses (AAV); Codon optimization; Liver gene therapy; Ornithine transcarbamylase deficiency (OTCD)

Indexed keywords

ADENOVIRUS VECTOR; AMMONIA; COMPLEMENTARY DNA; ORNITHINE CARBAMOYLTRANSFERASE; OROTIC ACID;

ANIMAL EXPERIMENT; ARTICLE; DRUG EFFICACY; ENZYME ACTIVITY; LIVER; MOUSE; NONHUMAN; ORNITHINE TRANSCARBAMYLASE DEFICIENCY; PRIORITY JOURNAL; PROTEIN EXPRESSION; URINALYSIS; URTICARIA; VIRAL GENE THERAPY;

ADULT; ANIMALS; DEPENDOVIRUS; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HUMANS; LIVER; MICE; ORNITHINE CARBAMOYLTRANSFERASE; ORNITHINE CARBAMOYLTRANSFERASE DEFICIENCY DISEASE; OROTIC ACID;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS - 8; MUS;

EID: 84856121122     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2011.10.020     Document Type: Article

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