Study of thrombopoietin for gene therapy of thrombocytopenia

Thrombopoietin (TPO) is likely to be a potent, specific and reliable medica tion in the treatment of thrombocytopenia. A TPO-highly-expressed plasmid p cDNA3-TPO was constructed and a primary study was made on the expression of TPO cDNA in vitro and gene transfer study for thrombocytopenia in vivo. rh TPO showed complete and stable bioactivity by a series of indicators. High expression of TPO was detected in plasma from healthy mice or thrombocytope nia mice model receiving direct intramuscular injection of pcDNA3-TPO. And the platelet level of healthy mice peaked to 1.9-fold of baseline. Mice wit h CTX-induced thrombocytopenia achieved profound nadirs, acceleration of re covery, 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 .