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Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease

Bishnu P. De,Jonathan B. Rosenberg,Nithya Selvan,Isabelle Wilson, Nadir Yusufzai,Alessandria Greco,Stephen M. Kaminsky,Linda A. Heier,Rodolfo J. Ricart Arbona,Ileana C. Miranda,Sebastien Monette,Anju Nair,Richie Khanna,Ronald G. Crystal,Dolan Sondhi

Human gene therapy（2023）

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关键词
CLN2,Batten,AAV,TPP-1,intracisternal delivery,nonhuman primates
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