Llwytho...
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...
Wedi'i Gadw mewn:
| Cyhoeddwyd yn: | Mol Genet Metab |
|---|---|
| Prif Awduron: | , , , , , , , , |
| Fformat: | Artigo |
| Iaith: | Inglês |
| Cyhoeddwyd: |
2017
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| 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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