Gene transfer for erythropoiesis enhancement

Mol Med Today. 1996 Aug;2(8):343-8. doi: 10.1016/1357-4310(96)81800-4.

Abstract

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.

Publication types

  • Review

MeSH terms

  • Anemia / therapy*
  • Animals
  • Cell Transplantation
  • Erythropoiesis*
  • Erythropoietin / administration & dosage
  • Erythropoietin / genetics*
  • Erythropoietin / metabolism
  • Gene Transfer Techniques*
  • Genetic Therapy*
  • Humans
  • Recombinant Proteins
  • beta-Thalassemia / therapy

Substances

  • Recombinant Proteins
  • Erythropoietin