Abstract:
: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.
journal_name
Acta Biochim Biophys Sin (Shanghai)journal_title
Acta biochimica et biophysica Sinicaauthors
Yang J,Liu X,Yu J,Sheng L,Shi Y,Li Z,Hu Y,Xue J,Wu L,Liang Y,Xia J,Liang Ddoi
10.1093/abbs/gmp080subject
Has Abstractpub_date
2009-12-01 00:00:00pages
1053-60issue
12eissn
1672-9145issn
1745-7270journal_volume
41pub_type
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