NIH VideoCasting

Our first speaker, Courtney Fitzhugh, is a Lasker Clinical Research Scholar in the NHLBI Laboratory of Early Sickle Mortality Prevention, where she explores new avenues of hematopoietic cell transplantation (HCT) for SCD and studies the long-term health effects of curative therapies. Dr. Fitzhugh will explain the risk–benefit perspective when considering haploidentical transplants for patients with sickle cell disease and also describe advances in reducing the high rate of graft rejection. Our second speaker, Ambroise Wonkam, is director of the McKusick-Nathans Institute and the Department of Genetic Medicine at Johns Hopkins Medicine. Dr. Wonkam sees genomics research accelerating the development of curative therapies for SCD in three ways: through targeting fetal hemoglobin (HbF), the strongest-known modifier of SCD clinical expression; through exploring RNA therapies, such as microRNA to target the production of HbF proteins, or messenger RNA mediating production of HbF or adult hemoglobin; and through identifying unknown genetic risk factors for SCD cardiovascular complications to address mortality, particularly in adults. The Demystifying Medicine Series, jointly sponsored by FAES and NIH, includes presentations on pathology, diagnosis, and therapy in the context of major disease problems and current research. Primarily directed toward PhD students, clinicians and program managers, this series is designed to help bridge the gap between advances in biology and their application to major human diseases. Each session includes clinical and basic science components presented by NIH staff and invitees. All students, trainees, fellows, and staff are welcome to participate.

For more information go to https://demystifyingmedicine.od.nih.gov