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Acta Biochimica et Biophysica Sinica Advance Access published online on September 28, 2009
Acta Biochimica et Biophysica Sinica, doi:10.1093/abbs/gmp080
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© The Author 2009. Published by ABBS Editorial Office in association with Oxford University Press on behalf of the Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences.

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

Junlin Yang, Xionghao Liu, Jiaoling Yu, Liang Sheng, Yan Shi, Zhuo Li, Youjin Hu, Jinfeng Xue, Lingqian Wu, Yu Liang, Jiahui Xia and Desheng Liang*

State Key Laboratory of Medical Genetics, Central South University, Changsha, China

* Correspondence address. Tel: +86-731-84805365; Fax: +86-731-84478152; E-mail: liangdesheng{at}sklmg.edu.cn


  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.

Keywords    Duchenne muscular dystrophy (DMD); gene therapy; gene targeting; non-viral vector; human ribosomal DNA locus; minidystrophin-GFP fusion gene

Received: June 29, 2009; Accepted: August 16, 2009
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