Sickle Cell Gene Therapy (Episode 170)

Primary Care Perspectives

New research has led to the approval of sickle cell gene therapy and CHOP has played a big role in both the research and implementation of this innovative treatment. In this episode, Dr. Abraham Haimed, an attending in the Division of Hematology at Children's Hospital of Philadelphia, explains how a patient's own hematopoietic stem cells can be edited to block the expression or function of a certain gene and reactivate normal fetal hemoglobin to cure sickle cell disease. While this treatment happens outside the primary care world, it is important for general pediatricians to understand how this treatment works and what to look out for after a patient receives gene therapy.