Liver-directed adeno-associated virus serotype 8 gene transfer rescues a lethal murine model of citrullinemia type 1

Gene Therapy

Volumn 20, Issue 12, 2013, Pages 1188-1191

  Chandler, R J ^a   Tarasenko, T N ^a   Cusmano Ozog, K ^b   Sun, Q ^c   Sutton, V R ^c   Venditti, C P ^a   McGuire, P J ^a  

^a NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^b CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^c BAYLOR COLLEGE OF MEDICINE   (United States)

Author keywords

AAV8; citrullinemia; hyperammonemia; urea cycle disorders

Indexed keywords

ADENOVIRUS VECTOR; AMMONIA; ARGININOSUCCINATE SYNTHASE; ARGININOSUCCINATE SYNTHASE 1; CITRULLINE; COMPLEMENTARY DNA; LIVER ENZYME; MESSENGER RNA; THYROXINE BINDING GLOBULIN; UNCLASSIFIED DRUG;

AMMONIA BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CITRULLINEMIA; CONTROLLED STUDY; DRUG EFFICACY; ENZYME ACTIVITY; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GROWTH CURVE; HAIR; MOUSE; MURINE MODEL; NONHUMAN; PRIORITY JOURNAL; REDUCTION; SURVIVAL; SYSTEMIC THERAPY; TARGET ORGAN; VIRAL GENE DELIVERY SYSTEM; WEIGHT GAIN;

ADENO-ASSOCIATED VIRUS; MURINAE; MUS;

AMMONIA; ANIMALS; ARGININOSUCCINATE SYNTHASE; CITRULLINE; CITRULLINEMIA; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LIVER; MICE; ORGAN SPECIFICITY; PHENOTYPE; THYROXINE-BINDING GLOBULIN;

EID: 84890120770     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2013.53     Document Type: Article

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