Development of gene therapy using bone-marrow-derived mesenchymal stem cells

利用骨髓间充质干细胞(MSCs)进行基因治疗技术的发展。首先，MSCs具有在肿瘤部位聚集的能力，我们用荷瘤的裸鼠研究了这种机制。结果，获得了重要的信息，当MSCs被用作癌症靶向基因治疗的平台时将是有用的。其次，为了确保MSCs基因操作的安全性，我们利用19号染色体上AAVS1基因座的腺相关病毒整合机制，对转基因的定点整合进行了基础研究。

The development of gene therapy technologies using mesenchymal stem cells(MSCs) was conducted. First, MSCs have the ability to accumulate at tumor sites, and we investigated the mechanisms using tumor-bearing nude mice. As a result, important information was obtained that will be useful when MSCs are utilized as a platform for cancer-targeted gene therapy. Second, in order to ensure the safety of genetic manipulation of MSCs, we conducted basic studies on the site-specific integration of transgenes using adeno-associated virus integration machinery for the AAVS1 locus on chromosome 19.