Curative therapy for sickle cell | SCAASF AEP 2024

We are joined by Dr. Mark Walters, a hematologist-oncologist who cares for children with blood disorders and cancer, and specializes in blood and bone marrow transplants. He is director of the Pediatric Blood and Marrow Transplant Program at UCSF Benioff Children's Hospital Oakland.

Walters' research focuses on transplant and gene therapy for sickle cell disease and thalassemia – which are both red blood cell disorders that involve abnormalities of hemoglobin (the protein in red blood cells that carries oxygen). His goal is to offer a curative therapy to every child he treats. Because finding a healthy donor for sickle cell disease can be difficult, he also investigates methods that aim to correct the patient's own blood system through gene therapy and return the edited blood cells to the patient's circulation.
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