title: Transgenic and gene knock-out mouse models of sickle cell anemia and the thalassemias.
abstract: Sickle cell anemia and the thalassemias are globally the most common class of inherited disorders. Current treatment options are limited (transfusion, iron chelation) and are not suited to large-scale use in developing countries where the population of affected individuals is expected to undergo a tremendous increase in the near future. As such, the development of more practical and more permanent therapies is urgently needed. Recently, transgenic and gene knock-out technologies have been used to create mouse models for sickle cell anemia and all of the clinically relevant thalassemias (hemoglobin Bart's hydrops fetalis, hemoglobin H disease, beta-thalassemia intermedia, beta-thalassemia major/Cooley's anemia). These newly developed murine models should play an important role in the development of improved approaches for treating these commonly occurring genetic diseases.
authors.affiliation: Human Genome Center, Lawrence Berkeley Laboratory, University of California, Berkeley 94720, USA.
authors.forename: C
authors.initials: C
authors.lastname: Pászty
mesh_terms: D000755:Anemia, Sickle Cell / Q000235:genetics; D000818:Animals; D004195:Disease Models, Animal; D051379:Mice; D018345:Mice, Knockout; D008822:Mice, Transgenic; D017086:beta-Thalassemia