A non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus

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 tar- geted a minidystrophin-GFP fusion gene into the hrDNA locus of HT1080 cells with a high site-specific integrated efficiency of 10-s, 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 hrDNAtargeting vector might be highly useful for DMD gene therapy study.