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Minimal Ureagenesis is Necessary for Survival in the Murine Model of Hyperargininemia Treated by AAV-based Gene Therapy
Hyperammonemia is less severe in arginase 1 deficiency compared with other urea cycle defects. Affected patients manifest hyperargininemia and infrequent episodes of hyperammonemia. Patients typically suffer from neurological impairment with cortical and pyramidal tract deterioration, spasticity, lo...
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| 發表在: | Gene Ther |
|---|---|
| Main Authors: | , , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
2014
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4320015/ https://ncbi.nlm.nih.gov/pubmed/25474440 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2014.106 |
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