Bone Marrow Transplantation for Genetic Diseases

Four recent editorials in the Journal have reviewed the potential for increasing the therapeutic armamentarium for genetic disease.^{1 2 3 4} Bone marrow transplantation has been considered in each of these editorials. Now, in this issue of the Journal, Lucarelli et al. present impressive data on the use of marrow transplantation for the treatment of β-thalassemia.⁵ In severely affected patients 8 to 15 years of age, functional engraftment as measured by correction of the hematologic abnormality has been produced. In the future, clinical success will be measured by continued freedom from hemolysis and the necessity of repeated transfusions, resulting in an eventual…