医学
遗传增强
内科学
相（物质）
疾病
基因
生物
物理
遗传学
量子力学
作者
Barry Greenberg,Matthew R.G. Taylor,Eric Adler,Steven D. Colan,David Ricks,Paul Yarabe,Pavan K. Battiprolu,Gaurav Y. Shah,Kinnari Patel,Matthew Coggins,Susanna Carou-Keenan,Jonathan D. Schwartz,Joseph W. Rossano
标识
DOI：10.1056/nejmoa2412392
摘要
A single infusion of RP-A501 appeared to be safe and was associated with cardiac LAMP2 expression and evidence of clinical improvement over a period of 24 to 54 months. (Funded by Rocket Pharmaceuticals; ClinicalTrials.gov number, NCT03882437.).
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(2025-6-4)
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