Published in
American Society for Clinical Investigation, Journal of Clinical Investigation, 10(133), 2023
DOI: 10.1172/jci164575
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Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies
Journal article published in 2023 by
Xin Chen 
,
Thomas Dong,
Yuhui Hu,
Raffaella De Pace ,
Rafael Mattera ,
Kathrin Eberhardt,
Marvin Ziegler ,
Terry Pirovolakis,
Mustafa Sahin ,
Juan S. Bonifacino ,
Darius Ebrahimi-Fakhari ,
Steven J. Gray
,
Thomas Dong,
Yuhui Hu,
Raffaella De Pace ,
Rafael Mattera ,
Kathrin Eberhardt,
Marvin Ziegler ,
Terry Pirovolakis,
Mustafa Sahin ,
Juan S. Bonifacino ,
Darius Ebrahimi-Fakhari ,
Steven J. Gray
This paper is made freely available by the publisher.
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