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Updated: May 12, 2026

An Ecdysone Receptor-based Singular Gene Switch for Deliberate Expression of Transgene with Robustness, Reversibility, and Negligible Leakiness
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An Ecdysone Receptor-based Singular Gene Switch for Deliberate Expression of Transgene with Robustness, Reversibility, and Negligible Leakiness

Published on: May 7, 2018

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一种拼接切换的寡核酸治疗改善了G6PCc.648G>T的小鼠中的1a类型的糖原储存疾病

Kentaro Ito1,2, Go Tajima2,3, Chikako Kamisato1

  • 1Specialty Medicine Research Laboratories I, Daiichi Sankyo Co., Ltd., Tokyo, Japan.

The Journal of clinical investigation
|October 3, 2023
PubMed
概括

一种新型的寡核酸疗法纠正了1a型 (GSD1a) 糖原储存疾病中的异常G6PC拼接,恢复了葡萄糖-6-酸酶-α活性,并在临床前模型中预防低血糖症.

关键词:
基因治疗是一种基因疗法.遗传性疾病是一种遗传性疾病.葡萄糖的新陈代谢.代谢过程中的代谢.治疗方法 治疗方法

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