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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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First in-human, intracisternal dosing of RGX-111, an investigational AAV gene therapy, for a 21-month-old child with mucopolysaccharidosis type I (MPS I): 3.5 year follow-up

Raymond Y. Wang, Nina Movsesyan,Shih-hsin Kan,Tammam Beydoun, Mery Taylor,Richard C. Chang,Dawn Phillips, Jenna Burke, Michelle Gilmor,Yoonjin Cho,Paulo Falabella,Laura Pisani

MOLECULAR GENETICS AND METABOLISM（2024）

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