GENE-TRANSFER FOR ERYTHROPOIESIS ENHANCEMENT

The spectrum of anemias treated with recombinant human erythropoietin is rapidly broadening. Lifelong treatment with very high doses is now under evaluation for beta-thalassemia and sickle cell anemia, These in dications make it worthwhile to search for methods that will allow a p ermanent systemic delivery of the hormone. Here, we review experimenta l gene-transfer-based procedures for erythropoietin delivery in vivo. In mice, both ex vivo and direct in vivo approaches for gene transfer have resulted in the long-term production of therapeutic levels of the hormone. Gene transfer of erythropoietin could become a viable altern ative to the injection of the purified recombinant protein once reliab le procedures for controlling transgene expression are available.