通过zwitterionic聚合物结合和融合,最大限度地降低CRISPR/Cas9系统的脱频率
Yanjiao Han1, Zhefan Yuan2, Sijin Luo Zhong2
1Molecular Engineering and Science Institute, University of Washington WA 98195 USA.
Chemical science
|June 16, 2023
概括
新的方法减少了CRISPR-Cas9基因组编辑中的非目标突变. Cas9与zwitterionic聚合物/的化学结合或基因融合可以最大限度地减少DNA不匹配,提高治疗安全性和应用.
科学领域:
- 分子生物学分子生物学
- 生物技术是生物技术.
- 基因编辑 基因编辑
背景情况:
- 聚类正规间隔短平行体重复 (CRISPR) 相关蛋白9 (Cas9) 系统是一种多功能基因组编辑工具.
- 由意外的DNA相互作用引起的非目标突变限制了CRISPR-Cas9的临床应用.
研究的目的:
- 在CRISPR-Cas9基因组编辑中开发新的策略,以尽量减少非目标突变.
- 为了提高CRISPR-Cas9技术的特异性和安全性,用于治疗.
主要方法:
- 在化学上对应Cas9与zwitterionicpCB聚合物.
- 在基因上将Cas9与zwitterionic (EK) 合在一起.
- 评估经过修改的CRISPR/Cas9核糖蛋白蛋白 (RNP) 的目标和非目标编辑效率.
主要成果:
- Zwitterlated或EKylated CRISPR/Cas9 RNPs显示显著减少了非目标DNA编辑.
- 目标编辑活动保持在与未经修改的CRISPR/Cas9.9相当的水平.
- 非目标效率平均降低了70%,在某些情况下降低了90%.
结论:
- 新型蛋白质和mRNA水平的修改有效地降低了非目标CRISPR-Cas9活动.
- 这些方法提供了一种简单而有效的方法来提高基因组编辑的安全性.
- 开发的策略有可能加速CRISPR-Cas9在生物学和医学中的应用.
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