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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
Main Authors: Morabito, Giuseppe, Giannelli, Serena G., Ordazzo, Gabriele, Bido, Simone, Castoldi, Valerio, Indrigo, Marzia, Cabassi, Tommaso, Cattaneo, Stefano, Luoni, Mirko, Cancellieri, Cinzia, Sessa, Alessandro, Bacigaluppi, Marco, Taverna, Stefano, Leocani, Letizia, Lanciego, José L., Broccoli, Vania
格式: Artigo
語言:Inglês
出版: American Society of Gene & Cell Therapy 2017
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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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