Liu Songlei, Striebel Johannes, Pasquini Giovanni, Ng Alex H M, Khoshakhlagh Parastoo, Church George M, Busskamp Volker
Department of Genetics, Blavatnik Institute, Harvard Medical School, Boston, MA, United States.
Wyss Institute for Biologically Inspired Engineering, Harvard University, Boston, MA, United States.
Front Genome Ed. 2021 Sep 1;3:715697. doi: 10.3389/fgeed.2021.715697. eCollection 2021.
Gene activation with the CRISPR-Cas system has great implications in studying gene function, controlling cellular behavior, and modulating disease progression. In this review, we survey recent studies on targeted gene activation and multiplexed screening for inducing neuronal differentiation using CRISPR-Cas transcriptional activation (CRISPRa) and open reading frame (ORF) expression. Critical technical parameters of CRISPRa and ORF-based strategies for neuronal programming are presented and discussed. In addition, recent progress on applications of CRISPRa to the nervous system are highlighted. Overall, CRISPRa represents a valuable addition to the experimental toolbox for neuronal cell-type programming.
利用CRISPR-Cas系统进行基因激活在研究基因功能、控制细胞行为以及调节疾病进展方面具有重大意义。在本综述中，我们调查了近期有关使用CRISPR-Cas转录激活（CRISPRa）和开放阅读框（ORF）表达进行靶向基因激活和多重筛选以诱导神经元分化的研究。介绍并讨论了基于CRISPRa和ORF的神经元编程策略的关键技术参数。此外，还重点介绍了CRISPRa在神经系统应用方面的最新进展。总体而言，CRISPRa是神经元细胞类型编程实验工具箱中的一项有价值的补充。
