Department of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN 46202, USA.
Mol Ther. 2024 Sep 4;32(9):2803-2806. doi: 10.1016/j.ymthe.2024.08.011. Epub 2024 Aug 19.
Genome editing technologies are rapidly evolving, from the early zinc-finger nucleases, transcription activator-like effector nucleases (TALENs), and CRISPR-Cas9 (Figure 1, initial genome editing technologies), which generate double-strand breaks (DSBs), to base editing, which makes precise nucleobase conversion without inducing DSBs, and prime editing, which can carry out all types of edits without DSBs or donor DNA templates. The emergence of these revolutionary technologies offers us unprecedented opportunities for biomedical research and therapy development.
基因组编辑技术正在迅速发展,从早期的锌指核酸酶、转录激活因子样效应物核酸酶(TALENs)和 CRISPR-Cas9(图 1,初始基因组编辑技术),它们产生双链断裂(DSBs),到碱基编辑,它可以在不诱导 DSB 的情况下进行精确的核碱基转换,以及 Prime 编辑,它可以在不使用 DSB 或供体 DNA 模板的情况下进行所有类型的编辑。这些革命性技术的出现为我们的生物医学研究和治疗开发提供了前所未有的机会。