Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies.
| Autor: | Xin Chen1, Dong, Thomas1, Yuhui Hu1, De Pace, Raffaella2, Mattera, Rafael2, Eberhardt, Kathrin3, Ziegler, Marvin3, Pirovolakis, Terry4, Sahin, Mustafa3, Bonifacino, Juan S.2, Ebrahimi-Fakhari, Darius3, Gray, Steven J.1 Steven.Gray@UTSouthwestern.edu |
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| Zdroj: | Journal of Clinical Investigation. 5/15/2023, Vol. 133 Issue 10, p1-16. 16p. |
| Databáze: | Academic Search Ultimate |
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