State Key Laboratory of Medical Genetics, Central South University, Changsha, China.
Acta Biochim Biophys Sin (Shanghai). 2009 Dec;41(12):1053-60. doi: 10.1093/abbs/gmp080.
Gene therapy has emerged as a promising approach for the lethal disorder of Duchenne muscular dystrophy (DMD). Using a novel non-viral delivery system, the human ribosomal DNA (hrDNA) targeting vector, we targeted a minidystrophin-GFP fusion gene into the hrDNA locus of HT1080 cells with a high site-specific integrated efficiency of 10(-5), in which the transgene could express efficiently and continuously. The minidystrophin-GFP fusion protein was easily found to localize on the plasma membrane of HT1080 cells, indicating its possible physiologic performance. Our findings showed that the hrDNA-targeting vector might be highly useful for DMD gene therapy study.
基因治疗已成为治疗杜氏肌营养不良症（DMD）这一致命疾病的一种有前途的方法。我们使用一种新型非病毒传递系统，即靶向人类核糖体 DNA（hrDNA）的载体，将一个微小肌营养不良蛋白-GFP 融合基因靶向整合到 HT1080 细胞的 hrDNA 基因座中，整合效率高达 10(-5)，其中转基因能够高效且持续地表达。很容易发现微小肌营养不良蛋白-GFP 融合蛋白定位于 HT1080 细胞的质膜上，表明其可能具有生理功能。我们的研究结果表明，靶向 hrDNA 的载体可能对 DMD 的基因治疗研究具有很高的应用价值。
