STEM CELL THERAPY OF CONGENITAL DEFECTS IN HEMATOPOIESIS

先天性造血缺陷的干细胞治疗

• 批准号: 6389508
• 负责人: DAVID G. NATHAN
• 金额: $ 171.72万
• 依托单位: DANA-FARBER CANCER INST
• 依托单位国家: 美国
• 财政年份: 1995
• 资助国家: 美国
• 起止时间: 1995-09-30 至 2005-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6389508
• 关键词: congenital blood disorder; gene therapy; hematopoiesis; hematopoietic stem cells; stem cell transplantation; transfection /expression vector

The overall goal of this SCOR proposal is to develop and conduct clinical trials to correct congenital diseases of the human hematopoietic stem cell. The central theme of the research plan is the development of new methods of cellular and transduction gene therapy for these diseases. Specifically, we will develop new methods for stem cell transplantation across HLA barriers and will attempt to treat Fanconi Anemia and Diamond-Blackfan Anemia by replacement gene therapy protocols for these diseases. Dr. Guinan's project will focus on new advances in the biological and clinical aspects of haplomismatch bone marrow transplantation. Several projects will focus on the basic biology of Fanconi Anemia and Diamond- Blackfan Anemia and on Retroviral Transfection Scale-Up and Phase I gene therapy protocols for bone marrow transplantation. Other projects will focus on the continued development of retroviral and lentiviral programs which fortify our clinical efforts. In Dr. Mulligan's project, the capacity of bone marrow stem cells to replace diseased muscle cells will be explored while Dr. Sodroski's project will investigate the adaptations of HIV that will permit the virus access to human cells. The strength of this proposal lies in its focused drive toward clinical experimentation while gathering data of basic importance and exploring new frontiers.

该SCOR提案的总体目标是开发和进行临床试验，以纠正人类造血干细胞的先天性疾病。该研究计划的中心主题是为这些疾病开发细胞和转导基因治疗的新方法。具体来说，我们将开发跨HLA屏障的干细胞移植新方法，并尝试通过替代基因治疗方案治疗范可尼贫血和钻石-布莱克范贫血。Guinan博士的项目将集中在单倍型不匹配骨髓移植的生物学和临床方面的新进展。有几个项目将侧重于范可尼贫血和钻石-布莱克范贫血的基础生物学以及逆转录病毒转染扩大规模和骨髓移植第一阶段基因治疗方案。其他项目将侧重于继续开发逆转录病毒和慢病毒项目，以加强我们的临床工作。在Mulligan博士的项目中，将探索骨髓干细胞替代患病肌肉细胞的能力，而Sodroski博士的项目将研究艾滋病毒的适应性，使病毒能够进入人类细胞。这一建议的优势在于它集中精力进行临床实验，同时收集具有基本重要性的数据并探索新的前沿。