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Updated: Sep 11, 2025

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传递信息:翻译mRNA治疗肝脏遗传代谢疾病的治疗方法

Sonam Gurung1,2, Dany Perocheau1, Roopkatha Ghosh1,2

  • 1Great Ormond Street Institute of Child Health, University College London, London, UK.

Journal of inherited metabolic disease
|August 19, 2025
PubMed
概括

传递 RNA (mRNA) 脂质纳米粒子 (LNP) 提供了一种革命性的基因治疗方法. 这种可适应的mRNA-LNP技术在治疗遗传代谢疾病方面表现有前途,目前正在进行临床试验,评估其安全性和有效性.

关键词:
基因治疗的基因疗法遗传代谢性疾病 遗传代谢性疾病脂质纳米颗粒是一种纳米粒子.肝脏 肝脏 肝脏 肝脏 肝脏 肝脏它们是mRNARNA.

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