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Updated: Sep 15, 2025

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
10:06

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells

Published on: April 26, 2017

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通过替代拼接来对致命的ERAD突变进行功能性救援

Huilun Helen Wang, Zhihong Wang, Liangguang Leo Lin

    bioRxiv : the preprint server for biology
    |July 15, 2025
    PubMed
    概括

    研究人员在ENDI综合征的小鼠模型中发现了一种天然的RNA拼接机制,该机制可以挽救内分泌网膜相关降解 (ERAD) 功能. 这一发现导致了反感性寡核酸 (ASO) 的开发,以恢复ERAD和蛋白质稳定,提供了潜在的治疗方法.

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