Hybrid nucleic acid molecules and vectors including amp;bgr;-globin regulatory elements

摘要

The invention relates to hybrid nucleic acid molecules for gene therapy in cells of the erythroid lineage, and in particular &agr;-, &bgr;-, &dgr;-, &egr;, &ggr;-, or &zgr;-globin nucleotide sequences operably linked to &bgr;-globin regulatory elements. The hybrid nucleic acid molecules, at single copy, are capable of producing a polypeptide. The hybrid nucleic acid molecules are useful for treatment of hemoglobinopathies such as sickle cell anemia or &bgr;-thalassemia.;The hybrid nucleic acid molecules are useful at single copy for erythroid expression at single copy of RNA and polypeptides in transgenic animals.