Our studies focus mainly on investigation of stem cell biology using the hematopoietic stem cell (HSC) as a research model. Recent identification of a variety of stem cell sources including embryonic and somatic (tissue-specific) stem cells has brought about substantial progress in the field of stem cell research. The HSC represents the first stem cell for which identity and existence were determined. Studies on HSCs have provided us with some basic concepts applying to different types of stem cells, yet many of these concepts remain unverified. It therefore is very important to continue basic studies to answer many questions left unsolved and thus to permit contributions to the field of biological research and clinical medicine. HSCs are capable of continuous supply of all lineages of blood cells to each individual for his or her entire life. Both self-renewal and multilineage differentiation potentials enable this task. One major advantage in HSC research lies in that established assay systems allow clonal analysis of each individual stem cell. Using a defined assay system, we can test capabilities of self-renewal and multilineage differentiation at single cell levels using either in vitro or in vivo assays. We believe that HSC research will eventually make great contributions to the development of safe and efficacious regenerative medicine and gene therapy.

Hematopoietic stem cells (HSCs) have great potential for use in gene therapy for blood disease patients. There are growing expectations that these next-generation medicines will meet the demand to treat rare diseases where small molecule drugs have not been successfully developed. To bring HSC gene therapy into practical use, the basics of HSC properties remain to be understood, and cell manufacturing processes, including cell isolation, expansion, and preservation, need to be developed. Additionally, a method to validate the quality and safety of the manufactured HSCs must be established. In our lab, we focus on the characteristics of each disease, explore the mechanisms, and determine the best-fit gene therapy for each disease entity. The information from these studies will be used to further develop manufacturing and validation processes for modified HSCs and other hematopoietic cells. We will also study unmet needs, which will lead us to develop new applications for HSCs.