RNA 和 DNA 的 RGEN 编辑：从催化 RNA 到 CRISPR 再到临床的漫长曲折之路。

RGEN Editing of RNA and DNA: The Long and Winding Road from Catalytic RNAs to CRISPR to the Clinic.

机构信息

Departments of Surgery, Pharmacology and Cancer Biology, and Biomedical Engineering, Duke University, 2 Genome Ct, Durham, NC 27710, USA.

出版信息

Cell. 2020 May 28;181(5):955-960. doi: 10.1016/j.cell.2020.04.050.

DOI:10.1016/j.cell.2020.04.050

PMID:32470403

Abstract

The first clinical studies utilizing RNA-guided endonucleases (RGENs) to therapeutically edit RNA and DNA in cancer patients were recently published. These groundbreaking technological advances promise to revolutionize genetic therapy and, as I discuss, represent the culmination of decades of innovative work to engineer RGENs for such editing applications.

摘要

最近发表了一些首次利用 RNA 引导的内切酶 (RGENs) 在癌症患者中治疗性编辑 RNA 和 DNA 的临床研究。这些开创性的技术进步有望彻底改变基因治疗，正如我所讨论的，它们代表了几十年来为这种编辑应用设计 RGENs 的创新工作的结晶。