AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spf(ash) Mice

Ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is associated with severe hyperammonemia accompanied by a high risk of neurological damage and death in patients presenting with the neonatal-onset form. Contemporary therapies, including liver transplantation, remain...

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Bibliografische gegevens
Hoofdauteurs: Cunningham, Sharon C, Spinoulas, Afroditi, Carpenter, Kevin H, Wilcken, Bridget, Kuchel, Philip W, Alexander, Ian E
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Nature Publishing Group 2009
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Original Articles
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835243/
https://ncbi.nlm.nih.gov/pubmed/19384294
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.88
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