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Induction and prevention of severe hyperammonemia in the spfash mouse model of ornithine transcarbamylase deficiency using shRNA and rAAV-mediated gene delivery.
Cunningham SC, Kok CY, Dane AP, Carpenter K, Kizana E, Kuchel PW, Alexander IE
Mol Ther. 2011 May;19(5):854-9. doi: 10.1038/mt.2011.32. Epub 2011 Mar 8.
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