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

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.

authors

Yang J,Liu X,Yu J,Sheng L,Shi Y,Li Z,Hu Y,Xue J,Wu L,Liang Y,Xia J,Liang D

doi

10.1093/abbs/gmp080

subject

Has Abstract

pub_date

2009-12-01 00:00:00

pages

1053-60

issue

12

eissn

1672-9145

issn

1745-7270

journal_volume

41

pub_type

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