Induction and prevention of severe hyperammonemia in the spf ash mouse model of ornithine transcarbamylase deficiency using shRNA and rAAV-mediated gene delivery

Molecular Therapy

Volumn 19, Issue 5, 2011, Pages 854-859

  Cunningham, Sharon C ^a   Kok, Cindy Y ^a   Dane, Allison P ^a   Carpenter, Kevin ^a,b   Kizana, Eddy ^c   Kuchel, Philip W ^b   Alexander, Ian E ^a,b  

^a CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^b UNIVERSITY OF SYDNEY   (Australia)

^c WESTMEAD HOSPITAL   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; MESSENGER RNA; ORNITHINE CARBAMOYLTRANSFERASE; PARVOVIRUS VECTOR; SHORT HAIRPIN RNA;

ACIDURIA; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; DOSE RESPONSE; DRUG DOSE REDUCTION; DRUG EFFICACY; ENZYME ACTIVITY; GENE CONSTRUCT; GENE SILENCING; GENE TARGETING; HUMAN; HUMAN CELL; HYPERAMMONEMIA; LOW DRUG DOSE; MOUSE; NEUROTOXICITY; NONHUMAN; ORNITHINE TRANSCARBAMYLASE DEFICIENCY; VIRAL GENE DELIVERY SYSTEM; VIRUS RECOMBINANT;

ADENO-ASSOCIATED VIRUS; MURINAE; MUS;

EID: 79955576172     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.32     Document Type: Article

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