FETAL MONKEY MODEL FOR GENE THERAPY FOR SICKLE CELL DISEASE

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目及 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 目的：镰状细胞病是基因治疗的第一个目标之一，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生能力。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的体内表达已被证明限于一小部分细胞，其不随时间持续。目的是探索提高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.