Bone marrow transplantation cures many blood disorders, but a major limitation is the scarcity of compatible blood forming (hematopoietic) stem cells and the challenges to maintain and expand these cells ex vivo for clinical use.
Blood-forming stem cells produce all the cells in the blood, including red blood cells, which carry oxygen to the tissues; white blood cells, which constitute the immune system of the body; and platelets, which participate in blood clotting and coagulation.
Investigators at the Black Family Stem Cell Institute focus on elucidating developmental, epigenetic, redox-mediated, and metabolic mechanisms that govern hematopoietic stem cell properties to be exploited for their maintenance and their expansion ex vivo. Ongoing clinical trials are attempting to translate some of these findings in patients.
Others focus on expanding cord blood hematopoietic stem cells or on generating new hematopoietic stem cells by differentiating patient-derived pluripotent stem cells or direct reprograming of patient cells for future transplantation therapies. These in vitro models of blood, including immune disorders, will help produce hematopoietic stem cells and overall blood, correcting genetic aberrations and identifying underlying mechanisms of diseased stem cells. We will also be able to use them for drug screening. Another promising line of investigation is dedicated to exploring mechanisms that promote aging of hematopoietic stem cells.
Aging is thought to lead to alterations of hematopoietic stem cell properties that increase the risk of blood cancers and immune deficiencies of the elderly. Ongoing efforts emphasize exposing processes involved in hematopoietic stem cell aging. Our overarching goal is to develop means to revert or delay blood stem cell aging for a longer, healthier life.
Investigators with a major focus in blood-forming stem cells include: