Naffakh N, Danos O
Laboratoire Rétrovirus et Transfert Génétique, Institut Pasteur, Paris, France.
Mol Med Today. 1996 Aug;2(8):343-8. doi: 10.1016/1357-4310(96)81800-4.
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 indications make it worthwhile to search for methods that will allow a permanent systemic delivery of the hormone. Here, we review experimental 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 alternative to the injection of the purified recombinant protein once reliable procedures for controlling transgene expression are available.
用重组人促红细胞生成素治疗的贫血症范围正在迅速扩大。目前正在评估对β地中海贫血和镰状细胞贫血进行终身大剂量治疗。这些适应症使得寻找能够实现激素全身性永久递送的方法变得很有价值。在此，我们综述了基于实验性基因转移的体内促红细胞生成素递送方法。在小鼠中，体外和直接体内基因转移方法均已实现激素治疗水平的长期产生。一旦有了控制转基因表达的可靠方法，促红细胞生成素基因转移可能成为注射纯化重组蛋白的可行替代方法。
