[Therapeutic perspectives of sickle cell anemia].
After a long gestation new therapies are at hand. At the present time about 30 patients have been cured with the bone marrow transplantation. The potential use of cord blood, rich in hematopoietic stem cells, is under development as an alternative for bone marrow transplantation. The activation of HbF expression upon hydroxyurea (Hydrea) and other agents which is under clinical trials. The agent 12C79 which increases the oxygen affinity of sickle cells in vivo and prevent HbS polymerization is in clinical development. Membrane acting agents which inhibit sickle cell dehydration are another approach to be developed. The inhibition of vascular cell adhesion, of inappropriate clotting formation and of inadequate response to vaso-occlusive events needs to be developed. Finally the gene therapy is a very promising avenue.
Beuzàrd
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Laboratoire de biochimie et INSERM U 91, CNRS UA607, hôpital Henri-Mondor, Créteil.
D000755:Anemia, Sickle Cell / Q000188:drug therapy / Q000601:surgery / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans
Perspectives thérapeutiques de la drépanocytose.