[Therapeutic perspectives of sickle cell anemia]. 1992

Y Beuzàrd
Laboratoire de biochimie et INSERM U 91, CNRS UA607, hôpital Henri-Mondor, Créteil.

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.

UI MeSH Term Description Entries
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000755 Anemia, Sickle Cell A disease characterized by chronic hemolytic anemia, episodic painful crises, and pathologic involvement of many organs. It is the clinical expression of homozygosity for hemoglobin S. Hemoglobin S Disease,HbS Disease,Sickle Cell Anemia,Sickle Cell Disease,Sickle Cell Disorders,Sickling Disorder Due to Hemoglobin S,Anemias, Sickle Cell,Cell Disease, Sickle,Cell Diseases, Sickle,Cell Disorder, Sickle,Cell Disorders, Sickle,Disease, Hemoglobin S,Hemoglobin S Diseases,Sickle Cell Anemias,Sickle Cell Diseases,Sickle Cell Disorder
D018380 Hematopoietic Stem Cell Transplantation Transfer of HEMATOPOIETIC STEM CELLS from BONE MARROW or BLOOD between individuals within the same species (TRANSPLANTATION, HOMOLOGOUS) or transfer within the same individual (TRANSPLANTATION, AUTOLOGOUS). Hematopoietic stem cell transplantation has been used as an alternative to BONE MARROW TRANSPLANTATION in the treatment of a variety of neoplasms. Stem Cell Transplantation, Hematopoietic,Transplantation, Hematopoietic Stem Cell

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