Study of thrombopoietin for gene therapy of thrombocytopenia
- 12 Downloads
Thrombopoietin (TPO) is likely to he a potent, specific and reliable medication in the treatment of thrombocytopenia. A TPO-highly-expressed plasmid pcDNA3-TPO was constructed and a primary study was made on the expression of TPO cDNAin vitro and gene transfer study for thrombocytopeniain vivo. rhTPO showed complete and stable bioactivity by a series of indicators. High expression of TPO was detected in plasma from healthy mice or thrombocytopenia mice model receiving direct intramuscular injection of pcDNA3-TPO. And the platelet level of healthy mice peaked to 1.9-fold of baseline. Mice with CTX-induced thrombocytopenia achieved profound nadirs, acceleration of recovery, even 1.8–2.0-fold supranormal levels of peripheral platelet counts. The results offered experimental support for clinical application of gene therapy for thrombocytopenia via direct intramuscular injection of TPO cDNA.
Keywordsthrombopoietin (TPO) gene transfer thrombocytopenia intramuscular injection
Unable to display preview. Download preview PDF.
- 13.Abina, M. A., Tulliez, M., Lacout, C. et al., Major effects of TPO delivered by a single injection of a recombinant aden-ovirus on prevention of septicemia and anemia associated with myelosuppression in mice: risk of sustained expression inducing myelofibrosis due to immunosuppression,Gene Ther., 1998, 5(4): 497.PubMedCrossRefGoogle Scholar