Hematopoietic Stem Cell Biology and Blood Disorders
The hematopoietic stem cell component of the Center of Regenerative Medicine at the Boston Medical Center/Boston University consists of a team of basic scientists and clinicians conducting state-of-the-art research centered around hematopoiesis, stem cell biology, and its application to disorders of the blood. The long term objectives of our work are to develop stem and cell-based therapies aimed at combating blood-borne diseases such as sickle cell anemia, amyloidosis, and various thrombotic disorders.
Background and Interests
Hematopoietic stem cells (HSC) are a rare and renewable population of cellswhich reside in the bone marrow of all mammals and are known to give rise to all of the differentiated cells of the adult peripheral blood. The HSC biology component of the CRM focuses upon the identification, study and manipulation of the stem cell compartment and resulting hematopoietic cells as developments borne in basic research are translated directly into therapies applied in the clinic.
In addition, recent conceptual and technical improvements have resulted in clinically meaningful levels of gene transfer into repopulating hematopoietic stem cells. We currently employ novel, highly efficient gene transfer strategies and methodologies at the CReM which have lead to new ways of studying and manipulating HSC, opening the door to possible human gene therapy approaches.
Lastly, the reprogramming of somatic cells into induced pluripotent stem (iPS) cells (Takahashi and Yamanaka, 2006) is a breakthrough discovery with the potential to be developed into a wide array of clinical applications. A variety of human somatic cells have been effectively reprogrammed, albeit at limited efficiencies, and their murine counterparts have been shown to have efficacy in the treatment of disease models. A major goal of the hematopoietic stem cell component of the CReM is the direct application of this technology to the study of the hematopoietic system and blood borne diseases. The iPS technology currently being developed in the group will allow for the creation of advanced disease models of the blood and may allow for the production of “custom” hematopoietic stem cells and fully differentiated hematopoietic cells for cell-based therapies that match the patient’s immunologic profile.