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AAV Gene Therapy Corrects OTC Deficiency and Prevents Liver Fibrosis in Aged OTC-Knock Out Heterozygous Mice

Ornithine transcarbamylase (OTC) deficiency is an X-linked disorder of the urea cycle. Hemizygous males and heterozygous females may experience life-threatening elevations of ammonia in blood and brain, leading to irreversible cognitive impairment, coma, and death. Recent evidence of acute liver fai...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Genet Metab
Prif Awduron: Wang, Lili, Bell, Peter, Morizono, Hiroki, He, Zhenning, Pumbo, Elena, Yu, Hongwei, White, John, Batshaw, Mark L., Wilson, James M.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: 2017
Pynciau:
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC5423267/
https://ncbi.nlm.nih.gov/pubmed/28283349
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.02.011
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