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 在此行政补充拨款申请中，我们请求资金购买新电池 分选仪（S3e™ 细胞分选仪，Bio-Rad）。这种最先进的细胞分选仪将使我们能够评估大量 大小基因敲入事件，包括靶向敲入、靶向插入/删除（indel） 在单细胞分辨率下，速率以及潜在的脱靶敲入和插入缺失事件。瞄准 1，我们计划使用该细胞分选仪来评估mi-spCas9-v2。在目标 2 中，我们将使用该细胞分选仪 评估 mi-saCas9。在目标3中，我们将使用该细胞分选仪来评估功效和安全性 miCas9 在临床相关应用中的概况。结果，这个新的细胞分选仪，一旦 配备，将产生更有说服力的数据来证明所提出的优点 miCas9 基因编辑核酸酶。