How Gene Therapies are Revolutionizing Care for Sickle Cell Disease

On this episode of Advances in Care, host Erin Welsh and Dr. Markus Mapara, Director of the Blood and Marrow Transplantation and Cell Therapy Program at NewYork-Presbyterian and Columbia, discuss the current challenges in treating sickle cell disease and how newly FDA approved gene editing treatments are leading to new treatment pathways. Dr. Mapara covers two new gene editing approaches, explaining that both involve editing stem cells and reintroducing them to patients’ bodies to curb the sickling of red blood cells. He also helps listeners to understand the difference between the two: one gene therapy uses CRISPR technology to help the body create fetal hemoglobin which mitigates cell sickling while the other uses a lentiviral vector to edit stem cells to produce anti-sickling hemoglobin that’s similar to fetal hemoglobin once reintroduced to the body. 

Dr. Mapara also goes into the innovative work that the Blood and Marrow Transplantation and Cell Therapy Program is doing with CAR-T cell therapies. And he shares how these advancements in treating sickle cell disease will have a significant impact on how physicians approach treating patients.

***

Dr. Mapara is a Professor of Medicine at Columbia University Medical Center and the Director of the Blood and Marrow Transplantation (BMT)/Cell Therapy Program at New York Presbyterian Hospital/Columbia University Medical Center. He specializes in the care of patients with sickle cell disease and certain blood cancers (Multiple Myeloma, Amyloidosis) undergoing bone marrow transplantation and gene therapy.  His research is focused on developing novel approaches to make bone marrow transplantation and cell therapy safer for patients.. 

For more information visit nyp.org/Advances