Journal
ACTA BIOCHIMICA ET BIOPHYSICA SINICA
Volume 41, Issue 12, Pages 1053-1060Publisher
OXFORD UNIV PRESS
DOI: 10.1093/abbs/gmp080
Keywords
Duchenne muscular dystrophy (DMD); gene therapy; gene targeting; non-viral vector; human ribosomal DNA locus; minidystrophin-GFP fusion gene
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Funding
- National Key Basic Research Program of China [2004CB518800]
- National High Technology Research and Development Program of China [2007AA021002, 2007AA021206]
- National Natural Science Foundation of China [30700458, 30971298]
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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.
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