FETAL MONKEY MODEL FOR GENE THERAPY FOR SICKLE CELL DISEASE

用于镰状细胞病基因治疗的胎猴模型

• 批准号: 7349628
• 负责人: YUET Wai KAN
• 金额: $ 2.48万
• 依托单位: UNIVERSITY OF CALIFORNIA AT DAVIS
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-05-01 至 2007-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7349628
• 关键词: FETAL; MONKEY; MODEL; GENE; THERAPY

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Objective: The long-term goal of these studies is to explore fetal gene transfer for sickle cell disease in a nonhuman primate model which closely simulates the human. Sickle cell disease was one of the first targets for gene therapy because the normal human globin genes are small and the target cell, the hematopoietic stem cell, has long-term repopulating capabilities. However, despite many advantages of globin gene transfer, the in vivo expression of the somatic globin transgene has been shown to be restricted to a small proportion of cells, which do not persist over time.

该子项目是利用 NIH/NCRR 资助的中心拨款提供的资源的众多研究子项目之一。子项目和研究者 (PI) 可能已从另一个 NIH 来源获得主要资金，因此可以在其他 CRISP 条目中得到体现。列出的机构是中心的机构，不一定是研究者的机构。目的：这些研究的长期目标是在非常模拟人类的非人灵长类动物模型中探索镰状细胞病的胎儿基因转移。镰状细胞病是基因治疗的首要目标之一，因为正常人类珠蛋白基因很小，而且靶细胞（造血干细胞）具有长期增殖能力。然而，尽管珠蛋白基因转移具有许多优点，但体细胞珠蛋白转基因的体内表达已被证明仅限于一小部分细胞，并且不会随着时间的推移而持续存在。