Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders

Building on CHOP's position as a global leader in cell and gene therapies, the Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (CuRED) program will develop and deliver novel gene therapies and stem cell transplants to patients in a family-centered clinic. Sickle cell disease (SCD) and beta thalassemia are devastating blood disorders that result in anemia, severe chronic pain, and premature death. Currently, treatment is largely limited to supportive care. The CuRED program aims to change the care pathways for red blood cell disorders.

Here are some highlights of the program's research efforts:

• Gene therapy of hemoglobinopathies (sickle cell, beta-thalassemia, and alpha-thalassemia)
• Gene therapy of congenital diserythropoietic and ring sideroblastic anemias (CDA and CSA)
• Gene therapy for metachromatic leukodystrophy (MLD)
• Non-toxic myeloablative regimens
• Erythropoiesis, iron metabolism, and inflammation.

The CuRED program received Frontier status for Fiscal Year 2020.