Gene Therapy for Sickle Cell Disease

Sickle cell disease is an inherited blood disorder that often causes debilitating pain and life-threatening complications. Many patients with the disease make dozens of ER visits a year, and must receive regular blood transfusions. And though recent breakthroughs in gene therapy are giving patients with sickle cell new hope – and new options – accessing these therapies can be challenging. In this episode of Breaking Through, cell and gene therapy expert Dr. Stephan Grupp and pediatric hematologist Dr. Alexis Thompson join Madeline to talk about how gene therapy for sickle cell disease can transform patients’ lives. They also discuss what teams at Children’s Hospital of Philadelphia (CHOP) are doing to ensure that these innovative therapies reach the children who need them most.