SBIR/STTR Award attributes
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.Project Narrative In this Administrative Supplement grant application, we request funds to purchase a new cell sorter (S3e™ Cell Sorter, Bio-Rad). This new equipment will enable us to generate convincing efficacy and safety data at the single cell resolution to demonstrate the advantages of the proposed miCas9 gene editing nucleases.