The Manton Center for Orphan Disease Research
George Q. Daley, MD, PhD
George Q. Daley, MD, PhD is renowned as one of the world's most innovative and accomplished stem cell scientists. His lab was first to prove that customized stem cell-based therapies can cure disease (in a mouse model of immune disorder), and he has pioneered cutting- edge techniques for creating stem cells a patient's body won't reject. These techniques, which include nuclear transfer and direct reprogramming, are key to unleashing stem cells' promise of conquering a broad sweep of debilitating and deadly disorders.
A specialist in cancers of the blood and bone marrow, his research contributed to the development of Gleevec, a targeted drug for the treatment of chronic myeloid leukemia. Dr. Daley and his team are now testing cell-based approaches in animal models of thalassemia, which will establish treatment principles for other inherited blood-based diseases such as sickle cell anemia and Fanconi anemia. The ultimate goal is to apply the knowledge learned in mouse models to build human models of disease, in order to correct problems at the genetic level and recreate new, healthy cells that can be used to restore or replace damaged tissues and organs.
Dr. Daley and his colleagues are making steady progress toward this goal. Using a tiny skin sample, Dr. Daley is able to reprogram the skin cells to produce pluripotent stem cells, i.e., cells that are capable of differentiating themselves into any tissue or organ in the body. These new cells, which are a blank slate, are used to create laboratory models of disease that can serve as a testing ground for gene repair methods. While such an approach is several years from being perfected, the possibility of using gene-based therapies have the potential to revolutionize the way that we treat a large number of orphan diseases. Once healthy brand-new cells are created, they can be put back into a child to assume their normal function.
Dr. Daley is president of the International Society for Stem Cell Research and a member of the Executive Committee of the Harvard Stem Cell Institute. A Howard Hughes Medical Institute Investigator, he recently received the Judson Daland Prize from the American Philosophical Society for outstanding achievements in patient-oriented clinical research.
|Congratulations to Dr. Daley for his recent appointment to the position of associate chief and director of Pediatric Stem Cell Transplantation in the Division of Hematology/Oncology at Children's. He will occupy the Samuel E. Lux IV Chair in Hematology and will oversee all aspects of the transplantation program.|
|Create your own stem cells in the Children's Virtual Stem Cell Laboratory!|