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: 7562145
• 负责人: YUET Wai KAN
• 金额: $ 2.46万
• 依托单位: UNIVERSITY OF CALIFORNIA AT DAVIS
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-05-01 至 2008-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7562145
• 关键词: Cells; Computer Retrieval of Information on Scientific Projects Database; Funding; Gene Expression; Gene Transfer; Genes; Globin; Grant; Hematopoietic stem cells; Human; Institution; Methods; Modeling; Monkeys; Research; Research Personnel; Resources; Sickle Cell Anemia; Source; Time; Transgenes; United States National Institutes of Health; fetal; gene therapy; in vivo

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: 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. The objective is to explore methods to enhance persistence and gene expression.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目和 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 目的：镰状细胞病是基因治疗的首要目标之一，因为正常人珠蛋白基因很小，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。目的是探索增强持久性和基因表达的方法。