FETAL MONKEY MODEL FOR GENE THERAPY FOR SICKLE CELL DISEASE

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。目的：这些研究的长期目标是探索在非人类灵长类动物模型中用于镰状细胞疾病的胎儿基因转移，该模型与人类非常相似。镰状细胞病是基因治疗的首批靶点之一，因为正常的人类珠蛋白基因很小，而靶细胞--造血干细胞--具有长期的再繁殖能力。然而，尽管珠蛋白基因转移有许多优点，但体细胞珠蛋白转基因在体内的表达已被证明仅限于一小部分细胞，这些细胞不会随着时间的推移而持续存在。

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.