Fetal Gene Therapy for Ornithine Transcarbamylase Deficiency by Intrahepatic Plasmid DNA-Micro-bubble Injection Combined with Hepatic Ultrasound Insonation
Abstract We evaluated the therapeutic efficacy of hepatic transfection of plasmid DNA using micro-bubbles and ultrasound insonation for fetal correction of ornithine transcarbamylase (OTC) deficiency in mice. Twenty-three sparse-fur heterozygous pregnant mice (day 16 of gestation) were divided into...
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| Veröffentlicht in: | Ultrasound in medicine & biology 2016-06, Vol.42 (6), p.1357-1361 |
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| creator | Oishi, Yoshie Kakimoto, Takashi Yuan, Wenji Kuno, Shuichi Yamashita, Hiromasa Chiba, Toshio |
| description | Abstract We evaluated the therapeutic efficacy of hepatic transfection of plasmid DNA using micro-bubbles and ultrasound insonation for fetal correction of ornithine transcarbamylase (OTC) deficiency in mice. Twenty-three sparse-fur heterozygous pregnant mice (day 16 of gestation) were divided into three groups: injection of plasmid-DNA micro-bubble mixture into fetal liver with ultrasound insonation (Tr, n = 11); control group 1 (C1), injection of plasmid-DNA micro-bubble mixture into fetal liver with no insonation (n = 5); and control group 2 (C2), injection of saline–micro-bubble mixture into fetal liver with ultrasound insonation (n = 7). Levels of blood ammonia and urinary orotic acid were significantly lower in the Tr group than in the C1 and C2 groups ( p < 0.05, p < 0.01, respectively), whereas OTC activity was not different between groups. Therefore, ultrasound insonation with micro-bubbles enhanced plasmid DNA transfection into fetal mouse liver, leading to one of the therapeutic methods in ammonia metabolism. This might provide more time for OTC-deficient infants until liver transplantation. |
| doi_str_mv | 10.1016/j.ultrasmedbio.2015.10.007 |
| format | Article |
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Twenty-three sparse-fur heterozygous pregnant mice (day 16 of gestation) were divided into three groups: injection of plasmid-DNA micro-bubble mixture into fetal liver with ultrasound insonation (Tr, n = 11); control group 1 (C1), injection of plasmid-DNA micro-bubble mixture into fetal liver with no insonation (n = 5); and control group 2 (C2), injection of saline–micro-bubble mixture into fetal liver with ultrasound insonation (n = 7). Levels of blood ammonia and urinary orotic acid were significantly lower in the Tr group than in the C1 and C2 groups ( p < 0.05, p < 0.01, respectively), whereas OTC activity was not different between groups. Therefore, ultrasound insonation with micro-bubbles enhanced plasmid DNA transfection into fetal mouse liver, leading to one of the therapeutic methods in ammonia metabolism. This might provide more time for OTC-deficient infants until liver transplantation.</description><identifier>ISSN: 0301-5629</identifier><identifier>EISSN: 1879-291X</identifier><identifier>DOI: 10.1016/j.ultrasmedbio.2015.10.007</identifier><identifier>PMID: 26995155</identifier><language>eng</language><publisher>England: Elsevier Inc</publisher><subject>Animals ; Animals, Newborn ; Disease Models, Animal ; Female ; Genetic Therapy - methods ; Liver ; Male ; Mice ; Mice, Inbred BALB C ; Micro-bubble ; Microbubbles ; Ornithine Carbamoyltransferase Deficiency Disease - embryology ; Ornithine Carbamoyltransferase Deficiency Disease - therapy ; OTC deficient mouse ; Plasmid DNA ; Plasmids - therapeutic use ; Prenatal gene therapy ; Radiology ; Sonotransfection ; Transfection - methods ; Ultrasonic Therapy - methods ; Ultrasound insonation</subject><ispartof>Ultrasound in medicine & biology, 2016-06, Vol.42 (6), p.1357-1361</ispartof><rights>World Federation for Ultrasound in Medicine & Biology</rights><rights>2016 World Federation for Ultrasound in Medicine & Biology</rights><rights>Copyright © 2016 World Federation for Ultrasound in Medicine & Biology. Published by Elsevier Inc. All rights reserved.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c468t-cc4ffb85e04e60274b358a2c0e767672bfd1ff9b2fc3d22872743997ce86873f3</citedby><cites>FETCH-LOGICAL-c468t-cc4ffb85e04e60274b358a2c0e767672bfd1ff9b2fc3d22872743997ce86873f3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://dx.doi.org/10.1016/j.ultrasmedbio.2015.10.007$$EHTML$$P50$$Gelsevier$$H</linktohtml><link.rule.ids>314,780,784,3550,27924,27925,45995</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/26995155$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Oishi, Yoshie</creatorcontrib><creatorcontrib>Kakimoto, Takashi</creatorcontrib><creatorcontrib>Yuan, Wenji</creatorcontrib><creatorcontrib>Kuno, Shuichi</creatorcontrib><creatorcontrib>Yamashita, Hiromasa</creatorcontrib><creatorcontrib>Chiba, Toshio</creatorcontrib><title>Fetal Gene Therapy for Ornithine Transcarbamylase Deficiency by Intrahepatic Plasmid DNA-Micro-bubble Injection Combined with Hepatic Ultrasound Insonation</title><title>Ultrasound in medicine & biology</title><addtitle>Ultrasound Med Biol</addtitle><description>Abstract We evaluated the therapeutic efficacy of hepatic transfection of plasmid DNA using micro-bubbles and ultrasound insonation for fetal correction of ornithine transcarbamylase (OTC) deficiency in mice. Twenty-three sparse-fur heterozygous pregnant mice (day 16 of gestation) were divided into three groups: injection of plasmid-DNA micro-bubble mixture into fetal liver with ultrasound insonation (Tr, n = 11); control group 1 (C1), injection of plasmid-DNA micro-bubble mixture into fetal liver with no insonation (n = 5); and control group 2 (C2), injection of saline–micro-bubble mixture into fetal liver with ultrasound insonation (n = 7). Levels of blood ammonia and urinary orotic acid were significantly lower in the Tr group than in the C1 and C2 groups ( p < 0.05, p < 0.01, respectively), whereas OTC activity was not different between groups. Therefore, ultrasound insonation with micro-bubbles enhanced plasmid DNA transfection into fetal mouse liver, leading to one of the therapeutic methods in ammonia metabolism. This might provide more time for OTC-deficient infants until liver transplantation.</description><subject>Animals</subject><subject>Animals, Newborn</subject><subject>Disease Models, Animal</subject><subject>Female</subject><subject>Genetic Therapy - methods</subject><subject>Liver</subject><subject>Male</subject><subject>Mice</subject><subject>Mice, Inbred BALB C</subject><subject>Micro-bubble</subject><subject>Microbubbles</subject><subject>Ornithine Carbamoyltransferase Deficiency Disease - embryology</subject><subject>Ornithine Carbamoyltransferase Deficiency Disease - therapy</subject><subject>OTC deficient mouse</subject><subject>Plasmid DNA</subject><subject>Plasmids - therapeutic use</subject><subject>Prenatal gene therapy</subject><subject>Radiology</subject><subject>Sonotransfection</subject><subject>Transfection - methods</subject><subject>Ultrasonic Therapy - methods</subject><subject>Ultrasound insonation</subject><issn>0301-5629</issn><issn>1879-291X</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2016</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqNks1u1DAUhSMEokPhFZDFik0G_yRxwgKpmqE_UqFItBI7y3auNQ6JPbUTUJ6Fl8XpDAixAXlhyfe759rnOMteEbwmmFRvuvXUj0HGAVpl_ZpiUqbCGmP-KFuRmjc5bciXx9kKM0zysqLNSfYsxg4nomL8aXZCq6YpSVmush_nMMoeXYADdLuDIPczMj6gm-DsuLPLaZAuahmUHOZeRkBbMFZbcHpGakZXLl1lB3s5Wo0-JWCwLdp-PMs_WB18rialekhUB3q03qGNH1SSbdH3pI8uj413Dw_yk2sTGr2TC_s8e2JkH-HFcT_N7s7f324u8-ubi6vN2XWui6oec60LY1RdAi6gwpQXipW1pBoDr9KiyrTEmEZRo1lLac0TwpqGa6irmjPDTrPXB9198PcTxFEMNmroe-nAT1GQGtdJiVL2b5TXvOCMYJzQtwc02RBjACP2wQ4yzIJgseQoOvFnjmLJcamllFLzy-OcSaXy79ZfwSVgewAgGfPNQhDxIRNobUhOi9bb_5vz7i8Z3Vtntey_wgyx81NwyXpBRKQCi8_Lj1o-FCkxLhtG2E-D4M0k</recordid><startdate>20160601</startdate><enddate>20160601</enddate><creator>Oishi, Yoshie</creator><creator>Kakimoto, Takashi</creator><creator>Yuan, Wenji</creator><creator>Kuno, Shuichi</creator><creator>Yamashita, Hiromasa</creator><creator>Chiba, Toshio</creator><general>Elsevier Inc</general><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>7X8</scope><scope>7QO</scope><scope>7TM</scope><scope>8FD</scope><scope>FR3</scope><scope>P64</scope></search><sort><creationdate>20160601</creationdate><title>Fetal Gene Therapy for Ornithine Transcarbamylase Deficiency by Intrahepatic Plasmid DNA-Micro-bubble Injection Combined with Hepatic Ultrasound Insonation</title><author>Oishi, Yoshie ; Kakimoto, Takashi ; Yuan, Wenji ; Kuno, Shuichi ; Yamashita, Hiromasa ; Chiba, Toshio</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c468t-cc4ffb85e04e60274b358a2c0e767672bfd1ff9b2fc3d22872743997ce86873f3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2016</creationdate><topic>Animals</topic><topic>Animals, Newborn</topic><topic>Disease Models, Animal</topic><topic>Female</topic><topic>Genetic Therapy - methods</topic><topic>Liver</topic><topic>Male</topic><topic>Mice</topic><topic>Mice, Inbred BALB C</topic><topic>Micro-bubble</topic><topic>Microbubbles</topic><topic>Ornithine Carbamoyltransferase Deficiency Disease - embryology</topic><topic>Ornithine Carbamoyltransferase Deficiency Disease - therapy</topic><topic>OTC deficient mouse</topic><topic>Plasmid DNA</topic><topic>Plasmids - therapeutic use</topic><topic>Prenatal gene therapy</topic><topic>Radiology</topic><topic>Sonotransfection</topic><topic>Transfection - methods</topic><topic>Ultrasonic Therapy - methods</topic><topic>Ultrasound insonation</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Oishi, Yoshie</creatorcontrib><creatorcontrib>Kakimoto, Takashi</creatorcontrib><creatorcontrib>Yuan, Wenji</creatorcontrib><creatorcontrib>Kuno, Shuichi</creatorcontrib><creatorcontrib>Yamashita, Hiromasa</creatorcontrib><creatorcontrib>Chiba, Toshio</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>Biotechnology Research Abstracts</collection><collection>Nucleic Acids Abstracts</collection><collection>Technology Research Database</collection><collection>Engineering Research Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><jtitle>Ultrasound in medicine & biology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Oishi, Yoshie</au><au>Kakimoto, Takashi</au><au>Yuan, Wenji</au><au>Kuno, Shuichi</au><au>Yamashita, Hiromasa</au><au>Chiba, Toshio</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Fetal Gene Therapy for Ornithine Transcarbamylase Deficiency by Intrahepatic Plasmid DNA-Micro-bubble Injection Combined with Hepatic Ultrasound Insonation</atitle><jtitle>Ultrasound in medicine & biology</jtitle><addtitle>Ultrasound Med Biol</addtitle><date>2016-06-01</date><risdate>2016</risdate><volume>42</volume><issue>6</issue><spage>1357</spage><epage>1361</epage><pages>1357-1361</pages><issn>0301-5629</issn><eissn>1879-291X</eissn><abstract>Abstract We evaluated the therapeutic efficacy of hepatic transfection of plasmid DNA using micro-bubbles and ultrasound insonation for fetal correction of ornithine transcarbamylase (OTC) deficiency in mice. Twenty-three sparse-fur heterozygous pregnant mice (day 16 of gestation) were divided into three groups: injection of plasmid-DNA micro-bubble mixture into fetal liver with ultrasound insonation (Tr, n = 11); control group 1 (C1), injection of plasmid-DNA micro-bubble mixture into fetal liver with no insonation (n = 5); and control group 2 (C2), injection of saline–micro-bubble mixture into fetal liver with ultrasound insonation (n = 7). Levels of blood ammonia and urinary orotic acid were significantly lower in the Tr group than in the C1 and C2 groups ( p < 0.05, p < 0.01, respectively), whereas OTC activity was not different between groups. Therefore, ultrasound insonation with micro-bubbles enhanced plasmid DNA transfection into fetal mouse liver, leading to one of the therapeutic methods in ammonia metabolism. This might provide more time for OTC-deficient infants until liver transplantation.</abstract><cop>England</cop><pub>Elsevier Inc</pub><pmid>26995155</pmid><doi>10.1016/j.ultrasmedbio.2015.10.007</doi><tpages>5</tpages></addata></record> |
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| subjects | Animals Animals, Newborn Disease Models, Animal Female Genetic Therapy - methods Liver Male Mice Mice, Inbred BALB C Micro-bubble Microbubbles Ornithine Carbamoyltransferase Deficiency Disease - embryology Ornithine Carbamoyltransferase Deficiency Disease - therapy OTC deficient mouse Plasmid DNA Plasmids - therapeutic use Prenatal gene therapy Radiology Sonotransfection Transfection - methods Ultrasonic Therapy - methods Ultrasound insonation |
| title | Fetal Gene Therapy for Ornithine Transcarbamylase Deficiency by Intrahepatic Plasmid DNA-Micro-bubble Injection Combined with Hepatic Ultrasound Insonation |
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