title: 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.affiliation: State Key Laboratory of Medical Genetics, Central South University, Changsha, China.
authors.forename: Junlin
authors.initials: J
authors.lastname: Yang
authors.forename: Xionghao
authors.initials: X
authors.lastname: Liu
authors.forename: Jiaoling
authors.initials: J
authors.lastname: Yu
authors.forename: Liang
authors.initials: L
authors.lastname: Sheng
authors.forename: Yan
authors.initials: Y
authors.lastname: Shi
authors.forename: Zhuo
authors.initials: Z
authors.lastname: Li
authors.forename: Youjin
authors.initials: Y
authors.lastname: Hu
authors.forename: Jinfeng
authors.initials: J
authors.lastname: Xue
authors.forename: Lingqian
authors.initials: L
authors.lastname: Wu
authors.forename: Yu
authors.initials: Y
authors.lastname: Liang
authors.forename: Jiahui
authors.initials: J
authors.lastname: Xia
authors.forename: Desheng
authors.initials: D
authors.lastname: Liang
mesh_terms: D015153:Blotting, Western; D004275:DNA, Ribosomal / Q000235:genetics*; D016189:Dystrophin / Q000235:genetics* / Q000378:metabolism; D015870:Gene Expression; D018390:Gene Targeting; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics*; D049452:Green Fluorescent Proteins / Q000235:genetics* / Q000378:metabolism; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000378:metabolism / Q000628:therapy*; D011993:Recombinant Fusion Proteins / Q000235:genetics / Q000378:metabolism; D020133:Reverse Transcriptase Polymerase Chain Reaction; D019076:Transgenes / Q000235:genetics; D014407:Tumor Cells, Cultured
chemical_list: D004275:DNA, Ribosomal; D016189:Dystrophin; D011993:Recombinant Fusion Proteins; D049452:Green Fluorescent Proteins