| 摘要 |
The invention relates to hybrid nucleic acid molecules for gene therapy in cells of the erythroid lineage, and in particular alpha-, beta-, delta-, epsi, gamma-, or zeta-globin nucleotide sequences operably linked to beta-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 beta-thalassemia.The hybrid nucleic acid molecules are useful at single copy for erythroid expression at single copy of RNA and polypeptides in transgenic animals.
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