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Updated: Jul 1, 2025

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针对β-thalassemia的RNA治疗药物

Hong-Quan Duong1, Thi-Hue Nguyen2, Minh-Cong Hoang3

  • 1Hanoi University of Public Health, Hanoi, Vietnam.

Progress in molecular biology and translational science
|March 8, 2024
PubMed
概括

像反感性寡核酸 (ASO) 这样的RNA疗法,通过纠正基因剪接来治疗β-thalassemia,显示出希望. 这些基于RNA的先进治疗方法旨在恢复患者的血红蛋白生产.

关键词:
这是一个很好的应用程序.反意义的寡核酸 (ASO)传递者RNA (miRNA) 是一种小RNA.对于RNA疗法来说,它是非常重要的.小型激活RNA (miRNA) 的使用在β-Thalassemia中,

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