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

通过间充质干细胞中突变基因的同源重组，开发腺病毒载体治疗大疱性表皮松解症的基因疗法。

• 批准号: 23791267
• 负责人: KIKUCHI Yasushi
• 金额: $ 2.66万
• 依托单位: Osaka University
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Young Scientists (B)
• 财政年份: 2011
• 资助国家: 日本
• 起止时间: 2011 至 2012
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/grant/KAKENHI-PROJECT-23791267/
• 关键词: 再生医療; 遺伝子治療; 表皮水疱症

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.

我们尝试了腺病毒载体介导的突变基因在间充质干细胞中的同源重组，用于大疱性表皮病的基因治疗。由于病毒转染的效率很低，我们被迫改变实验计划。然而，我们终于有了新的见解的基因表达谱以及信号转导的招募骨髓间充质干细胞。这些结果将有助于干细胞募集因子在再生医学中的应用。