FETAL MONKEY MODEL FOR GENE THERAPY FOR SICKLE CELL DISEASE

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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.