| 摘要 |
<p>The present invention provides a method for ameliorating β-globin disorders in a mammal. In one aspect of the invention, the treatment involves ex vivo treatment of early erythroid progenitor cells that leads to an increase in the relative amounts of cells subsequently expressing and accumulating HbF. The cell treatment is to be followed by transplantation of the modified cells. In another aspect of the invention, the same modification of progenitor cells occurs in vivo. Both treatments are based on the novel discovery that the modification can be performed very early in the erythroid maturation process, without disturbance of the subsequent proliferation and maturation of the erythroid precursor. The present invention also provides a procedure for the monitoring of β-globinopathies and the response of a patient to treatment. In this aspect of the invention, erythropoiesis of a patient is studied (in vivo or in vitro) by generating profiles of correlated contents of different types of hemoglobin present in nucleated red cells.</p> |
