Key Laboratory of Animal Genetics, Breeding and Reproduction of Shaanxi Province, College of Animal Science and Technology, Northwest A&F University, Yangling, Shaanxi, China; Central Laboratory, Changzhi Medical College, Changzhi, Shanxi, China.
Key Laboratory of Animal Genetics, Breeding and Reproduction of Shaanxi Province, College of Animal Science and Technology, Northwest A&F University, Yangling, Shaanxi, China.
J Biol Chem. 2022 Jul;298(7):102103. doi: 10.1016/j.jbc.2022.102103. Epub 2022 Jun 6.
Base editing has emerged as a revolutionary technology for single nucleotide modifications. The cytosine and adenine base editors (CBEs and ABEs) have demonstrated great potential in clinical and fundamental research. However, screening and isolating target-edited cells remains challenging. In the current study, we developed a universal Adenine and Cytosine Base-Editing Antibiotic Resistance Screening Reporter (ACBE-ARSR) for improving the editing efficiency. To develop the reporter, the CBE-ARSR was first constructed and shown to be capable of enriching cells for those that had undergone CBE editing activity. Then, the ACBE-ARSR was constructed and was further validated in the editing assays by four different CBEs and two versions of ABE at several different genomic loci. Our results demonstrated that ACBE-ARSR, compared to the reporter of transfection (RoT) screening strategy, improved the editing efficiency of CBE and ABE by 4.6- and 1.9-fold on average, respectively. We found the highest CBE and ABE editing efficiencies as enriched by ACBE-ARSR reached 90% and 88.7%. Moreover, we also demonstrated ACBE-ARSR could be employed for enhancing simultaneous multiplexed genome editing. In conclusion, both CBE and ABE activity can be improved significantly using our novel ACBE-ARSR screening strategy, which we believe will facilitate the development of base editors and their application in biomedical and fundamental research studies.
碱基编辑技术已经成为单核苷酸修饰的一项革命性技术。胞嘧啶碱基编辑器(CBEs)和腺嘌呤碱基编辑器(ABEs)在临床和基础研究中显示出巨大的潜力。然而,筛选和分离目标编辑细胞仍然具有挑战性。在本研究中,我们开发了一种通用的腺嘌呤和胞嘧啶碱基编辑抗生素抗性筛选报告基因(ACBE-ARSR),以提高编辑效率。为了开发该报告基因,首先构建了 CBE-ARSR,并证明其能够富集那些经历过 CBE 编辑活性的细胞。然后,构建了 ACBE-ARSR,并在几个不同的基因组位点,通过四种不同的 CBE 和两种版本的 ABE 在编辑实验中进一步验证。我们的结果表明,与转染报告(RoT)筛选策略相比,ACBE-ARSR 平均分别将 CBE 和 ABE 的编辑效率提高了 4.6 倍和 1.9 倍。我们发现,通过 ACBE-ARSR 富集的最高 CBE 和 ABE 编辑效率分别达到 90%和 88.7%。此外,我们还证明 ACBE-ARSR 可用于增强同时进行的多重基因组编辑。总之,我们的新型 ACBE-ARSR 筛选策略可显著提高 CBE 和 ABE 的活性,我们相信这将有助于碱基编辑器的发展及其在生物医学和基础研究中的应用。