Gene Therapy Approaches for Sickle Cell Anemia

Sickle cell disease (SCD) has a widely variable clinical course; some of them are severely affected in childhood while in others major damage does not manifest until adulthood. There is no test that can be used to predict the disease course. The best predictor of SCD severity is a patient ’s course to date [1]. Patients’ quality of life is low and life expectancy is relatively short, nearly 50 years, even newly developed treatments such as iron chelating and disease modifying agents. The only curative treatment option is allogeneic stem cell transplantation (Allo-SCT), but only o ne fifth of the patients have matched sibling donor.
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Source Type: research