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

我们通过骨髓间充质干细胞中突变基因的同源重组，尝试用腺病毒载体进行大疱性表皮松解症的基因治疗。由于病毒转染率很低，我们被迫改变了实验计划。然而，我们终于在基因表达谱以及从骨髓中招募间充质干细胞的信号转导方面有了新的见解。这些结果将有助于干细胞募集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.