N=1:

Jillian Belgrad1,2, Erin McConnell2,3, Stef Leonard2

  • 1RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA 01605, United States.

Nucleic acids research
|April 25, 2025
PubMed
概括

N=1协作 (N1C) 正在为个性化药物建立一个开放的生态系统,最初将重点放在罕见神经退行性疾病的抗意义寡核酸 (ASO) 上,以实现安全,可扩展的基因向疗法.

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