Adeno-associated virus-mediated rescue of neonatal lethality in argininosuccinate synthetase-deficient mice

Molecular Therapy

Volumn 21, Issue 10, 2013, Pages 1823-1831

  Kok, Cindy Y ^a   Cunningham, Sharon C ^a,b   Carpenter, Kevin H ^a,c   Dane, Allison P ^a   Siew, Susan M ^a   Logan, Grant J ^a   Kuchel, Philip W ^c   Alexander, Ian E ^a,c  

^a CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^b UNIVERSITY OF SYDNEY   (Australia)

^c UNIVERSITY OF SYDNEY   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

ARGININOSUCCINATE SYNTHASE; PARVOVIRUS VECTOR; VIRUS ANTIBODY;

ADULTHOOD; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY RESPONSE; ARTICLE; CITRULLINEMIA; CONTROLLED STUDY; DRUG EFFICACY; DRUG TARGETING; FEMALE; GENE EXPRESSION; GENE TRANSFER; HISTOPATHOLOGY; HUMAN; HUMAN CELL; HUMORAL IMMUNITY; HYPERAMMONEMIA; LIVER PARENCHYMA; MILK LEVEL; MOUSE; NEWBORN; NEWBORN MORTALITY; NONHUMAN; POSTNATAL CARE; PRENATAL CARE; RECURRENT DISEASE; SURVIVAL TIME; TRANSGENE; VIROTHERAPY;

ADENO-ASSOCIATED VIRUS; MUS;

ANIMALS; ANIMALS, NEWBORN; ARGININOSUCCINATE SYNTHASE; CITRULLINEMIA; DEPENDOVIRUS; FEMALE; FETAL THERAPIES; FETOSCOPY; GENETIC THERAPY; GENETIC VECTORS; HEK293 CELLS; HUMANS; HYPERAMMONEMIA; IMMUNITY, MATERNALLY-ACQUIRED; LIVER; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; ORGAN SPECIFICITY; PREGNANCY; TRANSGENES;

EID: 84885020193     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.139     Document Type: Article

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