Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA; Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, USA; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA; Department of Genetics, Cell Biology and Development, University of Minnesota, Minneapolis, Minnesota, USA.
Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, USA; Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, USA; Center for Genome Engineering, University of Minnesota, Minneapolis, Minnesota, USA; Stem Cell Institute, University of Minnesota, Minneapolis, Minnesota, USA.
Cytotherapy. 2023 Mar;25(3):270-276. doi: 10.1016/j.jcyt.2022.11.013. Epub 2023 Jan 10.
Consistent progress has been made to create more efficient and useful CRISPR-Cas9-based molecular toolsfor genomic modification.
This review focuses on recent articles that have employed base editors (BEs) for both clinical and research purposes.
CRISPR-Cas9 BEs are a useful system because of their highefficiency and broad applicability to gene correction and disruption. In addition, base editing has beensuggested as a safer approach than other CRISPR-Cas9-based systems, as it limits double-strand breaksduring multiplex gene knockout and does not require a toxic DNA donor molecule for genetic correction.
As such, numerous industry and academic groups are currently developing base editing strategies withclinical applications in cancer immunotherapy and gene therapy, which this review will discuss, with a focuson current and future applications of in vivo BE delivery.
在开发更高效、更有用的基于 CRISPR-Cas9 的基因组修饰分子工具方面，已经取得了持续的进展。
本综述重点关注了最近在临床和研究方面应用碱基编辑器 (BEs) 的文章。
CRISPR-Cas9 BE 是一种很有用的系统，因为其具有高效率和广泛的适用性，可用于基因纠正和破坏。此外，碱基编辑被认为比其他基于 CRISPR-Cas9 的系统更安全，因为它在多重基因敲除时限制双链断裂，并且不需要有毒的 DNA 供体分子进行基因纠正。
因此，许多工业和学术团体目前正在开发具有癌症免疫治疗和基因治疗临床应用的碱基编辑策略，本综述将讨论这方面的内容，重点介绍体内 BE 传递的当前和未来应用。
