A newly developed single-treatment approach, uses autologous gene therapy and CD34 stem cells to improve erythrocyte function in SSD patients.
Sickle cell disease (SSD) refers to a group of inherited erythrocyte disorders. Hemoglobin in people with SSD becomes defective leading to abnormal shaping of erythrocytes. The defective hemoglobin form, hemoglobin S, replaces the normal hemoglobin A. The abnormally crescent or sickle-shaped erythrocytes cause a number of illnesses associated with the clogging of vessels, poor oxygen flow to organs, and associated damage.