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AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy
The lack of technology for direct global-scale targeting of the adult mouse nervous system has hindered research on brain processing and dysfunctions. Currently, gene transfer is normally achieved by intraparenchymal viral injections, but these injections target a restricted brain area. Herein, we d...
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| 發表在: | Mol Ther |
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| Main Authors: | , , , , , , , , , , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
American Society of Gene & Cell Therapy
2017
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5768559/ https://ncbi.nlm.nih.gov/pubmed/28882452 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.08.004 |
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