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通过CRISPR/Cas9-或Cas12a-Mediated Knock-In在人类T细胞中提高基因编辑效率

Natalia Kruglova¹, Mikhail Shepelev¹

¹Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology RAS, 119334 Moscow, Russia.

|January 23, 2024

概括

使用CRISPR/Cas技术对T淋巴细胞的基因组编辑显示出癌症和艾滋病毒治疗的前景. 对CRISPR组件,传递和细胞培养的关键改进提高了临床应用的编辑效率,特别是在基因敲门中.

科学领域:

免疫学免疫学免疫学
分子生物学分子生物学
生物技术是生物技术.

背景情况:

T淋巴细胞对于适应性免疫是至关重要的,也是基因修饰的关键目标.
应用包括设计T细胞以向瘤或抵抗HIV等病毒感染.
由于CRISPR/Cas技术的可编程性和可访问性,它是T细胞基因组编辑的主要方法.

研究的目的:

审查影响人类T细胞编辑效率的关键参数.
专注于使CRISPR/Cas在T细胞中的临床应用成为可能的进展.
专门解决T细胞内基因敲门的挑战和成功.

主要方法:

审查CRISPR/Cas技术组件和交付系统.
对影响编辑结果的T细胞培养条件的分析.
检查提高基因编辑效率和特异性的策略.

主要成果:

最初的CRISPR/Cas在T细胞中的效率很低,阻碍了临床转化.
在CRISPR组件,传递方法和细胞培养方面取得了显著的改进.
优化的协议现在允许合理的编辑水平适合治疗用途,特别是基因敲门.

结论:

为了高效的T细胞基因组编辑,CRISPR/Cas技术已经得到了大量的改进.
这些进展为基于T细胞的免疫疗法和传染病治疗铺平了道路.
进一步优化,特别是基因敲进,对于更广泛的临床实施至关重要.

关键词:

卡特卡特卡特这就是CRISPR/CasPR.这就是HDR HDR.艾滋病毒基因治疗艾滋病毒基因治疗基因组编辑基因组编辑敲进来 - 敲进来主要的T细胞主要是T细胞.

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相关概念视频

CRISPR

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...