A non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus
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 tar- geted a minidystrophin-GFP fusion gene into the hrDNA locus of HT1080 cells with a high si...
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| Veröffentlicht in: | Acta biochimica et biophysica Sinica 2009-12, Vol.41 (12), p.1053-1060 |
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| container_title | Acta biochimica et biophysica Sinica |
| container_volume | 41 |
| creator | Yang, Junlin Liu, Xionghao Yu, Jiaoling Sheng, Liang Shi, Yan Li, Zhuo Hu, Youjin Xue, Jinfeng Wu, Lingqian Liang, Yu Xia, Jiahui Liang, Desheng |
| description | 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 tar- geted a minidystrophin-GFP fusion gene into the hrDNA locus of HT1080 cells with a high site-specific integrated efficiency of 10-s, 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 hrDNAtargeting vector might be highly useful for DMD gene therapy study. |
| doi_str_mv | 10.1093/abbs/gmp080 |
| format | Article |
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Using a novel non-viral delivery system, the human ribosomal DNA （hrDNA） targeting vector, we tar- geted a minidystrophin-GFP fusion gene into the hrDNA locus of HT1080 cells with a high site-specific integrated efficiency of 10-s, 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 hrDNAtargeting vector might be highly useful for DMD gene therapy study.</description><identifier>ISSN: 1672-9145</identifier><identifier>EISSN: 1745-7270</identifier><identifier>DOI: 10.1093/abbs/gmp080</identifier><identifier>PMID: 20011980</identifier><language>eng</language><publisher>China: Oxford University Press</publisher><subject>Blotting, Western ; DMD ; DNA, Ribosomal - genetics ; Dystrophin - genetics ; Dystrophin - metabolism ; Gene Expression ; Gene Targeting ; Genetic Therapy ; Genetic Vectors - genetics ; GFP ; Green Fluorescent Proteins - genetics ; Green Fluorescent Proteins - metabolism ; Humans ; Muscular Dystrophy, Duchenne - genetics ; Muscular Dystrophy, Duchenne - metabolism ; Muscular Dystrophy, Duchenne - therapy ; Recombinant Fusion Proteins - genetics ; Recombinant Fusion Proteins - metabolism ; Reverse Transcriptase Polymerase Chain Reaction ; Transgenes - genetics ; Tumor Cells, Cultured ; 基因定位 ; 基因治疗 ; 营养不良症 ; 蛋白基因 ; 非病毒载体</subject><ispartof>Acta biochimica et biophysica Sinica, 2009-12, Vol.41 (12), p.1053-1060</ispartof><rights>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. 2009</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c377t-31040b752a219b4e12fe5ac9b2a9071ff554b4e65c8a929f474e61c69d0991d63</citedby><cites>FETCH-LOGICAL-c377t-31040b752a219b4e12fe5ac9b2a9071ff554b4e65c8a929f474e61c69d0991d63</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Uhttp://image.cqvip.com/vip1000/qk/90160X/90160X.jpg</thumbnail><link.rule.ids>314,776,780,27903,27904</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/20011980$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Yang, Junlin</creatorcontrib><creatorcontrib>Liu, Xionghao</creatorcontrib><creatorcontrib>Yu, Jiaoling</creatorcontrib><creatorcontrib>Sheng, Liang</creatorcontrib><creatorcontrib>Shi, Yan</creatorcontrib><creatorcontrib>Li, Zhuo</creatorcontrib><creatorcontrib>Hu, Youjin</creatorcontrib><creatorcontrib>Xue, Jinfeng</creatorcontrib><creatorcontrib>Wu, Lingqian</creatorcontrib><creatorcontrib>Liang, Yu</creatorcontrib><creatorcontrib>Xia, Jiahui</creatorcontrib><creatorcontrib>Liang, Desheng</creatorcontrib><title>A non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus</title><title>Acta biochimica et biophysica Sinica</title><addtitle>Acta Biochimica et Biophysica Sinica</addtitle><description>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 tar- geted a minidystrophin-GFP fusion gene into the hrDNA locus of HT1080 cells with a high site-specific integrated efficiency of 10-s, 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 hrDNAtargeting vector might be highly useful for DMD gene therapy study.</description><subject>Blotting, Western</subject><subject>DMD</subject><subject>DNA, Ribosomal - genetics</subject><subject>Dystrophin - genetics</subject><subject>Dystrophin - metabolism</subject><subject>Gene Expression</subject><subject>Gene Targeting</subject><subject>Genetic Therapy</subject><subject>Genetic Vectors - genetics</subject><subject>GFP</subject><subject>Green Fluorescent Proteins - genetics</subject><subject>Green Fluorescent Proteins - metabolism</subject><subject>Humans</subject><subject>Muscular Dystrophy, Duchenne - genetics</subject><subject>Muscular Dystrophy, Duchenne - metabolism</subject><subject>Muscular Dystrophy, Duchenne - therapy</subject><subject>Recombinant Fusion Proteins - genetics</subject><subject>Recombinant Fusion Proteins - metabolism</subject><subject>Reverse Transcriptase Polymerase Chain Reaction</subject><subject>Transgenes - genetics</subject><subject>Tumor Cells, Cultured</subject><subject>基因定位</subject><subject>基因治疗</subject><subject>营养不良症</subject><subject>蛋白基因</subject><subject>非病毒载体</subject><issn>1672-9145</issn><issn>1745-7270</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2009</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkU1P3DAQhq2qCCjtqffK6qE9oIA_kjg-rtgClYD20J4tx7GzbhM72A5S-PV4tQu9lcNoPvTMO9K8AHzE6AwjTs9l28bzfpxQg96AY8zKqmCEobe5rhkpOC6rI_Auxj8I0brG6BAcEYQw5g06Bo8r6LwrHmyQA3zQKvkATY7JJ-2SzcP17Rr22mmYNjrIaYExzd0C2wUmGXqdrOuhhKN1tltiCn7aWFdcXf6EZo7Wu92udclvBeAmrO9WcPBqju_BgZFD1B_2-QT8vvz26-K6uPlx9f1idVMoylgqKEYlallFJMG8LTUmRldS8ZZIjhg2pqrKPK4r1UhOuClZbrCqeYc4x11NT8DXne4U_P2sYxKjjUoPg3Taz1EwShtcNlWTyS__JQmmiNGGZfB0B6rgYwzaiCnYUYZFYCS2poitKWJnSqY_7WXndtTdC_vswr-7fp5eUfq8v7vxrr_PrxetVH-NHbSgpOQ5GvoExWChKA</recordid><startdate>20091201</startdate><enddate>20091201</enddate><creator>Yang, Junlin</creator><creator>Liu, Xionghao</creator><creator>Yu, Jiaoling</creator><creator>Sheng, Liang</creator><creator>Shi, Yan</creator><creator>Li, Zhuo</creator><creator>Hu, Youjin</creator><creator>Xue, Jinfeng</creator><creator>Wu, Lingqian</creator><creator>Liang, Yu</creator><creator>Xia, Jiahui</creator><creator>Liang, Desheng</creator><general>Oxford University Press</general><scope>2RA</scope><scope>92L</scope><scope>CQIGP</scope><scope>W94</scope><scope>WU4</scope><scope>~WA</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7QO</scope><scope>7U9</scope><scope>8FD</scope><scope>FR3</scope><scope>H94</scope><scope>P64</scope><scope>RC3</scope><scope>7X8</scope></search><sort><creationdate>20091201</creationdate><title>A non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus</title><author>Yang, Junlin ; Liu, Xionghao ; Yu, Jiaoling ; Sheng, Liang ; Shi, Yan ; Li, Zhuo ; Hu, Youjin ; Xue, Jinfeng ; Wu, Lingqian ; Liang, Yu ; Xia, Jiahui ; Liang, Desheng</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c377t-31040b752a219b4e12fe5ac9b2a9071ff554b4e65c8a929f474e61c69d0991d63</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2009</creationdate><topic>Blotting, Western</topic><topic>DMD</topic><topic>DNA, Ribosomal - genetics</topic><topic>Dystrophin - genetics</topic><topic>Dystrophin - metabolism</topic><topic>Gene Expression</topic><topic>Gene Targeting</topic><topic>Genetic Therapy</topic><topic>Genetic Vectors - genetics</topic><topic>GFP</topic><topic>Green Fluorescent Proteins - genetics</topic><topic>Green Fluorescent Proteins - metabolism</topic><topic>Humans</topic><topic>Muscular Dystrophy, Duchenne - genetics</topic><topic>Muscular Dystrophy, Duchenne - metabolism</topic><topic>Muscular Dystrophy, Duchenne - therapy</topic><topic>Recombinant Fusion Proteins - genetics</topic><topic>Recombinant Fusion Proteins - metabolism</topic><topic>Reverse Transcriptase Polymerase Chain Reaction</topic><topic>Transgenes - genetics</topic><topic>Tumor Cells, Cultured</topic><topic>基因定位</topic><topic>基因治疗</topic><topic>营养不良症</topic><topic>蛋白基因</topic><topic>非病毒载体</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Yang, Junlin</creatorcontrib><creatorcontrib>Liu, Xionghao</creatorcontrib><creatorcontrib>Yu, Jiaoling</creatorcontrib><creatorcontrib>Sheng, Liang</creatorcontrib><creatorcontrib>Shi, Yan</creatorcontrib><creatorcontrib>Li, Zhuo</creatorcontrib><creatorcontrib>Hu, Youjin</creatorcontrib><creatorcontrib>Xue, Jinfeng</creatorcontrib><creatorcontrib>Wu, Lingqian</creatorcontrib><creatorcontrib>Liang, Yu</creatorcontrib><creatorcontrib>Xia, Jiahui</creatorcontrib><creatorcontrib>Liang, Desheng</creatorcontrib><collection>中文科技期刊数据库</collection><collection>中文科技期刊数据库-CALIS站点</collection><collection>中文科技期刊数据库-7.0平台</collection><collection>中文科技期刊数据库-自然科学</collection><collection>中文科技期刊数据库-自然科学-生物科学</collection><collection>中文科技期刊数据库- 镜像站点</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>Biotechnology Research Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>AIDS and Cancer Research Abstracts</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><collection>MEDLINE - Academic</collection><jtitle>Acta biochimica et biophysica Sinica</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Yang, Junlin</au><au>Liu, Xionghao</au><au>Yu, Jiaoling</au><au>Sheng, Liang</au><au>Shi, Yan</au><au>Li, Zhuo</au><au>Hu, Youjin</au><au>Xue, Jinfeng</au><au>Wu, Lingqian</au><au>Liang, Yu</au><au>Xia, Jiahui</au><au>Liang, Desheng</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>A non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus</atitle><jtitle>Acta biochimica et biophysica Sinica</jtitle><addtitle>Acta Biochimica et Biophysica Sinica</addtitle><date>2009-12-01</date><risdate>2009</risdate><volume>41</volume><issue>12</issue><spage>1053</spage><epage>1060</epage><pages>1053-1060</pages><issn>1672-9145</issn><eissn>1745-7270</eissn><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 tar- geted a minidystrophin-GFP fusion gene into the hrDNA locus of HT1080 cells with a high site-specific integrated efficiency of 10-s, 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 hrDNAtargeting vector might be highly useful for DMD gene therapy study.</abstract><cop>China</cop><pub>Oxford University Press</pub><pmid>20011980</pmid><doi>10.1093/abbs/gmp080</doi><tpages>8</tpages></addata></record> |
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| ispartof | Acta biochimica et biophysica Sinica, 2009-12, Vol.41 (12), p.1053-1060 |
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| subjects | Blotting, Western DMD DNA, Ribosomal - genetics Dystrophin - genetics Dystrophin - metabolism Gene Expression Gene Targeting Genetic Therapy Genetic Vectors - genetics GFP Green Fluorescent Proteins - genetics Green Fluorescent Proteins - metabolism Humans Muscular Dystrophy, Duchenne - genetics Muscular Dystrophy, Duchenne - metabolism Muscular Dystrophy, Duchenne - therapy Recombinant Fusion Proteins - genetics Recombinant Fusion Proteins - metabolism Reverse Transcriptase Polymerase Chain Reaction Transgenes - genetics Tumor Cells, Cultured 基因定位 基因治疗 营养不良症 蛋白基因 非病毒载体 |
| title | A non-viral vector for potential DMD gene therapy study by targeting a minidystrophin-GFP fusion gene into the hrDNA locus |
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