Gene Therapy: Sickle Cell Disease and Beyond

Gene Therapy: Sickle Cell Disease and Beyond


Advances in gene therapy and other cellular therapies offer promising new therapeutic options that have the potential to revolutionize care for many hematologic conditions, including hemophilia, beta thalassemia, and recently, sickle cell disease (SCD). After this series, hematologists will understand the pathophysiology and symptoms of SCD, current diagnostic and management approaches, and the fundamentals of gene therapy for hematological indications.