The in vivo gene expression profile of the rAAV-2-hGM-CSF, rAAV-2-mGM-CSF vector modified bone marrow mesenchymal stem cell

Objective: To observe the in vivo gene expression profile of the recombinant adenoassociated-2 virus mediated human GM-CSF, mouse GM-CSF (rAAV-2-hGM-CSF, rAAV-2-mGM-CSF) vector modified bone marrow mesenchymal stem cell (BMSC). Methods: We transduced the BMSC by rAAV-2-hGM-CSF, rAAV-2-mGM-CSF at the condition which have acquired before respectively, then transfused the in vitro gene modified BMSC after 12 days proliferation in vitro to 6 weeks old nude mice through tail vein, while the BMSC transfused in control group hadn' t been gene modified. 2, 4, 6, 8 weeks after transfusion, count the total white blood cells and detect the hGM-CSF, mGM-CSF concentration in nude mice serum at that time point. Results: Nude mice serum hGM-CSF levels were 23.77, 26.32, 19.77, 15.25 ng/L at 2, 4, 6, 8 weeks after transfusion compare to 36.25 ng/L, the in vitro level before transfusion; mGM-CSF levels were 34.96, 34.84, 35.50, 32.93 ng/L at 2, 4, 6, 8 weeks after transfusion compare to 25.14 ng/L, the in vitro level before transfusion; at the same time point the nude mice serum mGM-CSF levels were 17.34, 17.44, 14.68, 16.85 ng/L in control group. rAAV-2-mGM-CSF transduced BMSC made the nude mice white blood cell count increased, but no changes in nude mice white blood cell count at rAAV-2-hGM-CSF transduced BMSC and control group. Conclusion: BMSC as a gene therapy vehicle, it can be gene modified in vitro, then the gene modified BMSC could let the therapeutic gene to have therapeutic effects in vivo.