Novel methods to improve nuclease mediated homologous recombination, Administrative suppl

改进核酸酶介导的同源重组的新方法，行政补充

• 批准号: 10640401
• 负责人: Jifeng Zhang
• 金额: $ 31.33万
• 依托单位: ATGC, INC.
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-07-01 至 2023-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10640401
• 关键词: Administrative Supplement; Applications Grants; Cells; Data; Equipment; Event; Funding; Genes; Knock-in; Mediating; Methods; Resolution; Safety; clinically relevant; efficacy evaluation; homologous recombination; improved; insertion/deletion mutation; novel; nuclease

Project Abstract In this Administrative Supplement grant application, we request funds to purchase a new cell sorter (S3e™ Cell Sorter, Bio-Rad). This state of art cell sorter will enable us to evaluate large size gene knock-in events, including both on-target knock-in, on-target insertion/deletion (indel) rates as well as potential off-target knock-in and indel events, at the single cell resolution. In Aim 1, we plan to use this cell sorter to evaluate mi-spCas9-v2. In Aim 2, we will use this cell sorter to evalute mi-saCas9. In Aim 3, we will use this cell sorter to evaluate the efficacy and safety profiles of miCas9 in clinically relevant applications. As a results, this new cell sorter, once equipped, will generate more convincing data to demonstrate the advantages of the proposed miCas9 gene editing nucleases.

项目摘要 在这份行政补充拨款申请中，我们申请资金购买一个新的牢房 分类器(S3e™细胞分类器，Bio-Rad)。这种最先进的细胞分类器将使我们能够评估大型 大小基因敲入事件，包括靶标敲入、靶标插入/缺失(INDel) 在单个单元格分辨率下的比率以及潜在的偏离目标的敲入和插入事件。在AIM 1，我们计划使用这个细胞分类器来评估mi-spCas9-v2。在目标2中，我们将使用这个细胞分类器 以评估mi-saCas9。在目标3中，我们将使用该细胞分选器来评估疗效和安全性 MiCas9在临床相关应用中的应用概况因此，这种新的细胞分类器，曾经 配备，将产生更令人信服的数据，以证明拟议的优势 MiCas9基因编辑核酸酶。