Induction and Prevention of Severe Hyperammonemia in the spf(ash) Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery

Urea cycle defects presenting early in life with hyperammonemia remain difficult to treat and commonly necessitate liver transplantation. Gene therapy has the potential to prevent hyperammonemic episodes while awaiting liver transplantation, and possibly also to avert the need for transplantation al...

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Hlavní autoři: Cunningham, Sharon C, Kok, Cindy Y, Dane, Allison P, Carpenter, Kevin, Kizana, Eddy, Kuchel, Philip W, Alexander, Ian E
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2011
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3098641/
https://ncbi.nlm.nih.gov/pubmed/21386824
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.32
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