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 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.
Naffakh
N
N
Laboratoire Rétrovirus et Transfert Génétique, Institut Pasteur, Paris, France.
Danos
O
O
D000740:Anemia / Q000628:therapy*; D000818:Animals; D017690:Cell Transplantation; D004920:Erythropoiesis; D004921:Erythropoietin / Q000008:administration & dosage / Q000235:genetics* / Q000378:metabolism; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006801:Humans; D011994:Recombinant Proteins; D017086:beta-Thalassemia / Q000628:therapy
D011994:Recombinant Proteins; D004921:Erythropoietin