CIRM Disease Team Planning Award
Children born with sickle cell anemia (SCA), caused by a genetic defect in hemoglobin, have severe anemia and damage to virtually all the body organs: the damage begins in infancy, and is frequently fatal by early adulthood. This is one of the most common inherited diseases in the world: because of California's ethnic diversity it is relatively common here, often in under-served populations. Our research team is dedicated to the treatment and, when possible, cure of this devastating disease. We and others have shown that transplantation of blood stem cells from bone marrow can cure SCA, and this type of stem cell therapy also is used in treating other blood and genetic diseases. Unfortunately, not all individuals are cured after bone marrow transplantation, as this is a risky treatment. We have carried out pioneering work showing that blood from the umbilical cord ("cord blood") of a newborn sibling can be used to cure blood disease in the affected sibling. A significant part of our effort is devoted to discovering ways to improve and extend the use of cord blood for blood cell transplantation and make this treatment less risky. Most recently, we have found that the placenta itself is a rich source of blood stem cells, and possibly also of stem cells for tissues other than blood. We have developed a program of clinical research that is closely associated and integrated with lab research that supports investigations into ways to improve stem cell transplantation and make it available to more people.
Childrens Hospital Oakland Research Institute
Disease Team Planning