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用于in vivo基因组编辑的NiCas12b的表征

Yunqian Zhang¹, Jingjing Wei², Hongyan Wang^3,4

¹Shanghai Key Laboratory of Metabolic Remodeling and Health, Institute of Metabolism and Integrative Biology, State Key Laboratory of Genetic Engineering at School of Life Sciences, Fudan University, Shanghai, 200438, China.

Advanced science (Weinheim, Baden-Wurttemberg, Germany)

|July 30, 2024

概括

一个新的CRISPR-Cas12b系统,NiCas12b,在哺乳动物细胞中显示出高效的基因组编辑,并在小鼠中成功降低胆固醇. 这一发现为基因治疗和研究提供了一个有前途的新工具.

科学领域:

分子生物学分子生物学
基因编辑技术的技术
生物技术是生物技术.

背景情况:

CRISPR-Cas12b系统是基因组编辑的强大工具,但其在人类细胞和体内应用是有限的.
识别具有高活性和特异性的新型Cas12b核酶对于推进基因编辑疗法至关重要.

研究的目的:

在哺乳动物细胞中选 Cas12b 的基因组编辑活动.
评估已识别的Cas12b核酶在体内基因编辑中的治疗潜力.

主要方法:

一种绿色光蛋白 (GFP) 激活试验被用来选15个Cas12b的ortologs.
NiCas12b通过腺相关病毒 (AAV) 传递,以准小鼠肝脏中的Pcsk9基因.
测量了插入/删除 (indel) 效率和血清胆固醇水平.

主要成果:

在15个选的Cas12b正义学家中,有4个在哺乳动物细胞中表现出编辑活性.
来自Nitrospira sp.的NiCas12b证明了高效的基因组编辑,具有"TTN"原空间器相邻动机 (PAM) 和高特异性.
在体内输送的NiCas12b导致小鼠肝脏的效率超过16.0%,显著降低血清胆固醇水平.

结论:

尼卡斯12b是一种高效和特定的CRISPR-Cas12b核酶,适合治疗应用.
这项研究验证了NiCas12b作为基础研究和临床基因编辑的新工具,特别是在诸如高胆固醇血等疾病中.

关键词:

这就是CRISPR-Cas.在NiCas12bb中使用.这就是PAMAM.在PCSK9中基因组编辑基因组编辑

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CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...