Evaluating Gene Therapy for Sickle Cell Disease: Advantages and Barriers
Description
Gene therapy is redefining the treatment landscape for sickle cell disease, with approaches like gene addition, gene editing, and fetal hemoglobin induction showing promise in reducing or even eliminating symptoms. However, while these therapies offer some advantages over allogeneic stem cell transplant, they also come with significant challenges. In a conversation with Ryan Quigley, Dr. David Jacobsohn discusses considerations for these therapeutic approaches and their potential impacts on care plans for sickle cell disease patients. Dr. Jacobsohn is the Division Chief of the Blood and Marrow Transplantation Program at Children’s National Hospital and Full Professor of Pediatrics at the George Washington University in Washington, DC.