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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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| Main Authors: | , , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2011
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| Assuntos: | |
| Acesso em linha: | 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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