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排名必读论文期刊顶会热点AI 简史溯源树小脉星探人才迁徙塔尖人才国防情报追踪开源工具智慧手语
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Phase 1/2 open-label, multi-center study to assess the safety, tolerability and efficacy of a single dose of PBGM01 delivered into the cisterna magna of subjects with type 1 (early onset) and type 2a (late onset) infantile GM1 gangliosidosis

Jeanine R. Jarnes,Debra L. Day-Salvatore,Can Ficicioglu,Caroline Hastings,Genevieve Bernard,Julien Baruteau,Fatih Ezgu,Roberto Giugliani,Michal Inbar-Feigenberg,Chester B. Whitley, Yan Ni,Victoria L. Ballard,Thomas F. Haws, David A. Weinstein

MOLECULAR GENETICS AND METABOLISM（2022）

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