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通过深度突变扫描和结构分析获得的基于AsCas12f的紧基因组编辑工具

Tomohiro Hino1, Satoshi N Omura2, Ryoya Nakagawa2

  • 1Department of Cardiovascular Medicine, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, Kyoto 602-8566, Japan.

Cell
|September 30, 2023
PubMed
概括

在人类细胞和小鼠中,设计的紧Cas12f变体 (enAsCas12f) 显示出增强的基因组编辑. 这些变种适用于腺相关病毒 (AAV) 传递,为体内基因治疗提供最小的平台.

关键词:
在CRISPR-Cas中动物实验化电磁场深度突变扫描基因疗法基因组编辑一个 iPS 单元

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