Gene therapy and bone marrow stem-cell transfer to treat autoimmune disease.

Current treatment of human autoimmune disease by autologous bone marrow stem-cell transfer is hampered by frequent disease relapses. This is most probably owing to re-emergent self-reactive lymphocytes. Gene therapy combined with bone marrow stem cells has successfully introduced genes lacking in immunodeficiences. Because the bone marrow compartment has a key role in establishing immune tolerance, this combination strategy should offer a rational approach to prevent re-emergent self-reactive lymphocytes by establishing solid, life-long immune tolerance to causative self-antigen. Indeed, we have recently demonstrated the success of this combination approach to prevent and cure an experimental autoimmune disease. We suggest that this combination strategy has the potential for translation to treat human autoimmune diseases in which causative self-antigens are known.