Gene Therapy: Hemoglobinopathies

Hemoglobin is a protein found in red blood cells that functions in the transport of oxygen from the lungs to the tissues and of carbon dioxide from the tissues to the lungs. Hemoglobinopathies include all genetic diseases of hemoglobin and are caused by genetic defects in the genes that express the hemoglobin protein. Worldwide, the most common forms of the condition are sickle-cell disease (SCD) and thalassemias. Several complications result from these genetic conditions, the effects of which can range from mild to severe. Until recently, the treatment of hemoglobinopathies has been limited to palliative care or allogeneic bone marrow transplantation; the latter has a high risk of graft-versus-host disease. Improved gene transduction and the development of induced pluripotent stem cells (iPSCs) have encouraged researchers ...