Improving iPSC reprogramming and CRISPR gene editing workflows and efficacy using CellRaft technology

使用 CellRaft 技术改进 iPSC 重编程和 CRISPR 基因编辑工作流程和功效

• 批准号: 10324993
• 负责人: Jessica Hartman
• 金额: $ 25.66万
• 依托单位: CELL MICROSYSTEMS, INC.
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-08-01 至 2023-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10324993
• 关键词: Academia; Adherent Culture; Automation; Capital; Case Study; Cell Culture Techniques; Cell Line; Cell Separation; Cell Survival; Cells; Cellular Morphology; Clinical; Clinical Trials; Clone Cells; Clustered Regularly Interspaced Short Palindromic Repeats; Consumption; Custom; DNA Sequence Alteration; Dermal; Development; Disease; Dissociation; Electroporation; Ensure; Equipment; Evaluation; Fibroblasts; Genes; Genetic; Harvest; Human; Image; Industry; Investments; Laboratories; Maintenance; Manuals; Measures; Methodology; Methods; Microscope; Microscopic; Monitor; Morphology; Nature; Pain; Patients; Phase; Phenotype; Plague; Polystyrenes; Population; Process; Protocols documentation; Publishing; Research Personnel; Seminal; Sickle Cell Anemia; Sorting - Cell Movement; Stains; System; Techniques; Technology; Therapeutic; Time; Tissues; Viral; Visualization; base; beta Thalassemia; cell immortalization; cell type; cost; cost effective; cost efficient; culture plates; drug development; drug discovery; feeding; gene therapy; improved; individual patient; induced pluripotent stem cell; instrumentation; microsystems; next generation sequencing; personalized medicine; pluripotency; precision drugs; programs; stem cell growth; stem cells; success; tool

Project Summary Induced pluripotent stem cells (iPSCs) have become a commonly used tool to generate multiple cell types from a given individual patient. Reprogramming non-invasively harvested cells, such as dermal fibroblasts, into iPSCs, allows investigators to generate any cell type from a patient with known genetic and clinical backgrounds. This method has been particularly powerful for cases in which a disease condition manifests in a tissue where acquiring patient-derived primary cells is challenging, or there are genetic mutations. However, the workflows associated with reprogramming, CRISPR gene editing, and differentiating iPSCs are low-throughput, costly, time-consuming, and not supported by commercially available automated instrumentation. Customized automation systems have been published and generally require considerable capital investment, multiple types of equipment, custom programming, and commitment to a single reprogramming method. There is an unmet need in academia and industry for technology that can improve the efficiency and success rates of a) reprogramming, b) iPSC viability, and c) the ability to generate clonally derived CRISPR edited cell lines in an automated fashion. The development of streamlined workflows on a single platform that can deliver higher efficiencies in iPSC reprogramming and cell line development is likely to dramatically enhance the utility and throughput of the technology, thereby accelerating the use of iPSCs in personalized medicine and drug discovery. Cell Microsystems proposes the use of the CellRaft Technology, comprising the automated AIR System and CytoSort Array cell culture consumable as a cost-effective, automated, and gentle solution that can solve many of the pain points that plague these critical workflows. In this Phase I program, we will develop protocols for the culture, reprogramming, and editing of iPSCs using our proprietary consumable and instrumentation and demonstrate that our technology provides faster, more cost effective, and more efficient workflows for iPSC cloning. Commercially, there is a tremendous potential market in laboratories across all industries that perform these workflows, and our company has a proven successful track record in developing tools for the drug discovery market.

项目总结