Adeno-associated virus-mediated delivery of erythropoietin leads to sustained elevation of hematocrit in nonhuman primates

Gene Ther. 1998 May;5(5):665-70. doi: 10.1038/


Recombinant adeno-associated virus encoding the monkey erythropoietin gene (rAAV-cm-Epo) was generated and tested for its potential to confer long-term expression of the gene product following intramuscular injection. A single intramuscular injection of 2 x 10(12) rAAV-cm-Epo particles into two baboons led to sustained high circulating Epo levels and a concomitant increase in hematocrit. The hematocrits reached 62 and 75% by week 10 (from pre-injection values of 38 and 40%, respectively) and remained elevated throughout the study period (28 weeks). Circulating Epo levels were also elevated throughout the study period. Our data demonstrate the potential for long-term gene expression in large animals by a single intramuscular injection of a recombinant adeno-associated virus (rAAV) vector.

MeSH terms

  • Animals
  • Antibodies / analysis
  • Dependovirus / genetics*
  • Enzyme-Linked Immunosorbent Assay
  • Erythropoietin / blood
  • Erythropoietin / genetics*
  • Fibrosarcoma
  • Gene Transfer Techniques*
  • Hematocrit
  • Humans
  • Injections, Intramuscular
  • Mice
  • Mice, Inbred BALB C
  • Mice, Inbred C57BL
  • Papio / immunology
  • Recombinant Proteins / blood
  • Time Factors


  • Antibodies
  • Recombinant Proteins
  • Erythropoietin