Disease Focus: Blood Disorders
Generation of functional cells and organs from iPSCs
The development of induced pluripotent stem cell (iPSC) technology may be the most important advance in stem cell biology for the future of medicine. This technology allows one to generate a patient’s own pluripotent stem cells (PSCs) from skin or blood cells. iPSCs can then be reprogrammed to multiply and produce high quality mature cells […]
Mechanisms of Hematopoietic stem cell Specification and Self-Renewal
During an individual’s lifetime, blood-forming cells in the bone marrow called hematopoietic stem cells (HSCs) supply all the red and white blood cells needed to sustain life. These blood stem cells are unique because they can make an identical copy of themselves (self-renew). Disorders of the blood system can be terminal, but such diseases may […]
Curing Hematological Diseases
The primary aim of this project is to develop treatments for incurable diseases of the blood and immune system. X-linked Severe Combined Immunodeficiency (X-SCID) and Fanconi anemia (FA) are two blood diseases where mutations in a single gene results in the disease. XSCID, more commonly known as the “bubble boy” disease, is characterized by a […]
Role of Innate Immunity in hematopoeitic stem cell-mediated allograft tolerance
The research proposed in this project has very high potential to identify new medications to boost the natural ability of stem cells to prevent rejection of transplanted organs. This is a very important goal, because patients that receive a life-saving transplanted organ must take toxic medications that increase their risk for cancer and serious infections. […]
Inactivating NK cell reactivity to facilitate transplantation of stem cell derived tissue
One of the great promises of stem cell research is that it will one day be possible to prepare replacement cells or organs from stem cells such as embryonic stem cells, which can be transplanted to patients to substitute for diseased or defective patient tissues or organs. Unfortunately, the immune system reacts against, and rejects, […]
Maternal and Fetal Immune Responses to In Utero Hematopoietic Stem Cell Transplantation
The immune system is the body’s defense system against disease and can recognize foreign cells. Because of this, stem cells and organs that are transplanted from one person to another are usually “rejected” by the immune system, forcing doctors to use powerful immune suppressive drugs with severe side effects. This natural defense system will therefore […]
Purified allogeneic hematopoietic stem cells as a platform for tolerance induction
Blood and immune cells originate and mature in the bone marrow. Bone marrow cells are mixtures of blood cells at different stages of development, and include rare populations of blood-forming stem cells. These stem cells are the only cells capable of generating the blood system for the life of an individual. Bone marrow transplants (BMT) […]
Development of Induced Pluripotent Stem Cells for Modeling Human Disease
Human embryonic stem cells (hESC) hold great promise in regenerative medicine and cell replacement therapies because of their unique ability to self-renew and their developmental potential to form all cell lineages in the body. Traditional techniques for generating hESC rely on surplus IVF embryos and are incompatible with the generation of genetically diverse, patient or […]
Prospective isolation of hESC-derived hematopoietic and cardiomyocyte stem cells
The capacity of human embryonic stem cells (hESCs) to perpetuate themselves indefinitely in culture and to differentiate to all cell types of the body has lead to numerous studies that aim to isolate therapeutically relevant cells for the benefit of patients, and also to study how genetic diseases develop. However, hESCs can cause tumors called […]
Improving microenvironments to promote hematopoietic stem cell development from human embryonic stem cells
Hematopoietic stem cells (HSC) have been used successfully to cure various life-threatening blood diseases. Yet, matching HSCs are not available for every patient. Human embryonic stem cells (hESC) may provide an unlimited source of HSCs for therapeutic use. However, hESC derived hematopoietic cells do not develop properly in those culture conditions that are currently used, […]