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Development of platforms for sorting, production, editing of beta cells

开发用于分类、生产、编辑 β 细胞的平台

基本信息

批准号：
10682155
负责人：
Amit Choudhary
金额：
$ 69.15万
依托单位：
BROAD INSTITUTE, INC.
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-09-05 至 2028-04-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10682155
关键词：
Affinity Anti-Inflammatory Agents Benchmarking Beta Cell Binding Biology CRISPR/Cas technology Cadaver Cell Culture Techniques Cell Line Cell Membrane Permeability Cell Proliferation Cell Survival Cell Transplantation Cell physiology Cell secretion Cells Chemicals Clinical Trials Dependence Development Dyes Encapsulated Endocrine Engineering Exhibits Face Fibrosis Foreign-Body Reaction Generations Genes Genetic Genome engineering Glucose Graft Rejection Harmine Human Immune Immune system Immunosuppressive Agents Impairment Insulin Insulin-Dependent Diabetes Mellitus Interleukin-10 Ions Islets of Langerhans Islets of Langerhans Transplantation Knock-in Legal patent Libraries Life Ligands Mitogens Pancreas Patients Plasma Population Prevention Procedures Prodrugs Production Proliferating Property Protein Tyrosine Kinase Protocols documentation Quality of life Replacement Therapy Reporting Scheme Sorting Source Specificity Surgical Replantation System Teratoma Therapeutic Therapeutic Effect Time Transplantation Treatment Cost Treatment Efficacy Undifferentiated United States National Institutes of Health Validation Zinc cell type chemical conjugate cost cost effective cytokine differentiation protocol elastase inhibitor embryonic stem cell engineered beta cell fluorophore genome editing glycemic control immunoregulation implantation in vivo induced pluripotent stem cell inhibitor islet large scale production mouse model precise genome editing side effect small molecule stem cells therapeutic development transplantation therapy

项目摘要

PROJECT SUMMARY Patients suffering from type 1 diabetes must undergo burdensome, often lifelong, exogenous insulin dependence. Up to now, the only available replacement therapy is to transplant islets from cadaveric donors. However, such procedures present hurdles such as the scarcity of available donors and the rejection of transplanted cells by the patient’s immune system. Also, over time, the transplanted islets tend to die. Several types of stem cells, including embryonic stem cell and induced pluripotent stem cell, have been extensively studied to generate glucose-responsive insulin-secreting cells and represents a more tenable source of islets. However, a significant concern for transplantation therapy is the need for immunosuppressive drugs, which exhibit long term side effects and hinders the feasibility of this approach to the large population of T1D patients. Furthermore, cost-effective and large-scale production of these stem cell-derived beta cells is still a major challenge. We propose to apply chemical biology and genome engineering approaches to develop platforms for efficient sorting, production, and editing of these beta cells. PHS 398/2590 (Rev. 06/09) 1 Continuation Format Page

项目总结