近红外光激活的圆形寡核酸用于对小鼠基因沉默的时空控制
Rong Wang1, Wen Chen2, Qiu Hong Qu1
1State Key Laboratory of Chemo and Bio-Sensing, College of Chemistry and Chemical Engineering, Hunan University, Changsha, 410082, China.
Angewandte Chemie (International ed. in English)
|December 9, 2025
概括
这项研究引入了一种新的近红外光可激活的圆形寡核酸平台,用于精确的基因调节. 这项技术可以在细胞和体内进行按需的基因沉默,为基因治疗提供一种更安全的方法.
科学领域:
- 分子生物学分子生物学
- 生物技术是生物技术.
- 治疗方法 治疗方法
背景情况:
- 寡核酸治疗药物为基因调节提供了潜在的潜力.
- 在体内实现精确的核酸功能时空控制仍然是一个挑战.
研究的目的:
- 开发一种新的近红外 (NIR) 光可激活的圆形寡核酸平台.
- 为了实现治疗应用的基因沉默的时空控制.
主要方法:
- 工程造型的圆形DNA酶和反感性寡核化物 (ASO) 具有NIR光分裂链接器.
- 通过NIR光照射触发左侧裂并激活寡核酸的功能.
- 在体外 (活细胞) 和体内 (瘤抑制) 的研究.
主要成果:
- 循环DNAzymes和ASOs显示最小的基底活性,但被NIR光有效地激活,以实现强大的基因沉默.
- 通过NIR激活的循环DNA酶在体内显示局部瘤抑制,具有最小的脱效应.
- 成功地实现了外源和内源mRNAs的基因沉默.
结论:
- 开发的平台在体内为寡核酸功能提供了多功能和有效的时空控制.
- 这项技术是基因功能研究的强大工具.
- 突出了开发安全,按需基因治疗的潜力.
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