Sustained Correction of OTC Deficiency in Spf (ash) mice Using Optimized Self-complementary AAV2/8 Vectors

Ornithine transcarbamylase deficiency (OTCD) is the most common inborn error of urea synthesis. Complete OTCD can result in hyperammonemic coma in the neonatal period which can rapidly become fatal. Current acute therapy involves dialysis; chronic therapy involves the stimulation of alternate nitrog...

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Autori principali: Wang, Lili, Wang, Huan, Morizono, Hiroki, Bell, Peter, Jones, David, Lin, Jianping, McMenamin, Deirdre, Yu, Hongwei, Batshaw, Mark L., Wilson, James M.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2011
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3321078/
https://ncbi.nlm.nih.gov/pubmed/21850052
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2011.111
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