da Silva Joana Ferreira, Tou Connor J, King Emily M, Eller Madeline L, Ma Linyuan, Rufino-Ramos David, Kleinstiver Benjamin P
Center for Genomic Medicine, Massachusetts General Hospital, Boston, MA, 02114, USA.
Department of Pathology, Massachusetts General Hospital, Boston, MA, 02114, USA.
bioRxiv. 2023 Sep 13:2023.09.12.557440. doi: 10.1101/2023.09.12.557440.
Genome editing technologies that install diverse edits can widely enable genetic studies and new therapeutics. Here we develop click editing, a genome writing platform that couples the advantageous properties of DNA-dependent DNA polymerases with RNA-programmable nickases (e.g. CRISPR-Cas) to permit the installation of a range of edits including substitutions, insertions, and deletions. Click editors (CEs) leverage the "click"-like bioconjugation ability of HUH endonucleases (HUHes) with single stranded DNA substrates to covalently tether "click DNA" (clkDNA) templates encoding user-specifiable edits at targeted genomic loci. Through iterative optimization of the modular components of CEs (DNA polymerase and HUHe orthologs, architectural modifications, etc.) and their clkDNAs (template configurations, repair evading substitutions, etc.), we demonstrate the ability to install precise genome edits with minimal indels and no unwanted byproduct insertions. Since clkDNAs can be ordered as simple DNA oligonucleotides for cents per base, it is possible to screen many different clkDNA parameters rapidly and inexpensively to maximize edit efficiency. Together, click editing is a precise and highly versatile platform for modifying genomes with a simple workflow and broad utility across diverse biological applications.
能够进行多种编辑的基因组编辑技术可广泛推动基因研究和新型治疗方法的发展。在此,我们开发了点击编辑技术,这是一种基因组书写平台,它将依赖DNA的DNA聚合酶的优势特性与RNA可编程切口酶(如CRISPR-Cas)相结合,以实现一系列编辑的安装,包括替换、插入和缺失。点击编辑器(CE)利用HUH核酸内切酶(HUHes)与单链DNA底物的“点击”样生物共轭能力,将编码用户可指定编辑的“点击DNA”(clkDNA)模板共价连接到目标基因组位点。通过对CE的模块化组件(DNA聚合酶和HUHe直系同源物、结构修饰等)及其clkDNA(模板配置、修复规避替换等)进行迭代优化,我们展示了以最小的插入缺失和无不需要的副产物插入来安装精确基因组编辑的能力。由于clkDNA可以按每个碱基几分钱的价格订购为简单的DNA寡核苷酸,因此可以快速且廉价地筛选许多不同的clkDNA参数,以最大化编辑效率。总之,点击编辑是一个精确且高度通用的平台,用于通过简单的工作流程和在各种生物学应用中的广泛用途来修饰基因组。