FETAL MONKEY MODEL FOR GENE THERAPY FOR SICKLE CELL DISEASE

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 目的：镰状细胞病是基因治疗的首选靶点之一，因为人类正常的珠蛋白基因很小，靶细胞--造血干细胞具有长期再繁殖的能力。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。其目的是探索增强持久性和基因表达的方法。

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.