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Updated: Jul 5, 2025

Removal of an Internal Translational Start Site from mRNA While Retaining Expression of the Full-Length Protein
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Published on: March 16, 2022

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RNA干扰效应器选择性地沉默致病变体GNAO1c.607 G > A In Vitro选择性地沉默致病变体

Natalia V Klementieva1,2, Evgenii A Lunev1,2,3, Anna A Shmidt1,2,3

  • 1Laboratory of Modeling and Gene Therapy of Hereditary Diseases, Institute of Gene Biology Russian Academy of Sciences, Moscow, Russia.

Nucleic acid therapeutics
|January 12, 2024
PubMed
概括

RNA干扰 (RNAi) 疗法可以针对主要的遗传疾病. 这项研究开发了RNAi工具来抑制导致GNAO1脑病变的GNAO1变体,显示出成功的等位基因歧视和突变蛋白减少.

关键词:
GNAO1脑病变是一种脑病变.这是一种RNA干扰.对于RNA疗法来说,它是一种治疗方法.腺相关病毒载体的病毒载体.基因抑制 基因抑制矢量化RNAi是指一个向量化的RNAi.

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