Large-scale generation of human red blood cells for transfusion therapy

描述（由申请人提供）：使用逆转录病毒基因转导方法，我们已经能够从原代人脐带血干细胞中产生条件永生化的长期造血干细胞系。该项目的第一阶段包括应用新发现的非病毒技术来产生人类细胞系，并随后将这些细胞分化为人类红细胞。此外，该项目还包括在小动物身上测试这些细胞的功能。该项目的第二阶段将需要优化蛋白转导的长期造血干细胞的大规模生产和红细胞的大规模生产，以便最终在大型动物和人类临床试验中进行测试。 公共卫生相关性：该项目旨在优化一种新的方法，从人类成体干细胞中产生大量用于临床的人类红细胞。一旦这种方法被验证用于大规模生产，造福公众健康的长期目标将是使用这种系统来产生红细胞，用于各种全球健康状况的临床应用，包括但不限于血液疾病，手术和急性创伤。

DESCRIPTION (provided by applicant): Using a method of retroviral gene transduction we have been able to generate conditionally immortalized long-term hematopoietic stem cell lines from primary human cord blood stem cells. Phase I of this project includes applying newly discovered non- viral technology to generate human cell lines and the subsequent differentiation of those cells into human red blood cells. In addition, the project includes testing the function of these cells in small animals. Phase II of the project will entail optimizing large-scale production of protein transduced long-term hematopoietic stem cells and large-scale production of red blood cells for eventual testing in larger animals and human clinical trials. PUBLIC HEALTH RELEVANCE: This project seeks to optimize a novel method to generate large amounts of human red blood cells for clinical use from human adult stem cells. Once this method has been validated for large-scale production, the long-term goal for benefiting public health will be to use this system to generate red blood cells for clinical applications across a variety of global health conditions including, but not limited to, blood disorders, surgeries and in cases of acute trauma.