Development of gene therapy for epidermolysis bullosa with adenoviral vector by homologous recombination of mutant gene in mesenchymal stem cells.

我们尝试了用腺病毒载体对间充质干细胞突变基因同源重组治疗大疱性表皮松解症。由于病毒转染的效率很低，我们被迫改变了实验计划。然而，我们最终对骨髓间充质干细胞募集的基因表达谱和信号转导有了新的认识。这些结果将有助于干细胞招募因子在再生医学中的应用。

We have tried the gene therapy for epidermolysis bullosa with adenoviral vector by homologous recombination of mutant gene in mesenchymal stem cells. As the efficiency of viral transfection was very low, we were forced to change the experimental plans. However, we finally had novel insights of gene expression profiles as well as signal transduction for the recruitment of mesenchymal stem cells from bone marrow. These results would be helpful for the application of regenerative medicine by stem cell-recruiting factors.