Gene transfer for erythropoiesis enhancement

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