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Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques
Mucopolysaccharidosis type I is a recessive genetic disease caused by deficiency of the lysosomal enzyme α-L-iduronidase, which leads to a neurodegenerative and systemic disease called Hurler syndrome in its most severe form. Several clinical trials are evaluating adeno-associated virus serotype 9 (...
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| 發表在: | Mol Ther Methods Clin Dev |
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| Main Authors: | , , , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
American Society of Gene & Cell Therapy
2018
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6070681/ https://ncbi.nlm.nih.gov/pubmed/30073179 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.06.003 |
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