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Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies.
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- Author(s): Chen, Xin; Dong, Thomas; Hu, Yuhui; De Pace, Raffaella; Mattera, Rafael; Eberhardt, Kathrin; Ziegler, Marvin; Pirovolakis, Terry; Sahin, Mustafa; Bonifacino, Juan S.; Ebrahimi-Fakhari, Darius; Gray, Steven J.
- Source:
Journal of Clinical Investigation. May 15, 2023, Vol. 133 Issue 10.
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