{"title":"利用asialal糖蛋白受体的肝脏选择性核酸靶向","authors":"T. Fukuma, George Y Wu, Catherine H. Wu","doi":"10.1163/156855800744548","DOIUrl":null,"url":null,"abstract":"Gene transfer to the liver by receptor-mediated endocytosis represents a promising method for therapeutic intervention of genetic disorders and acquired diseases affecting the liver. Based on the observation that asialoglycoprotein receptors are abundantly expressed on the surface of human hepatocytes in vivo, gene delivery and expression by DNA-ligand complexes targeted to liver have been developed in several different experimental models. This review will cover general aspects related to targeting to the liver via the asialoglycoprotein receptor, and the relationship between gene expression, persistence, the structure and size of DNA complexes, as well as the current strategies aimed to improve the overall efficiency of receptor-mediated gene therapy.","PeriodicalId":93646,"journal":{"name":"Gene therapy and regulation","volume":"1 1","pages":"79-93"},"PeriodicalIF":0.0000,"publicationDate":"2000-03-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1163/156855800744548","citationCount":"4","resultStr":"{\"title\":\"Liver-selective nucleic acid targeting using the asialoglycoprotein receptor\",\"authors\":\"T. Fukuma, George Y Wu, Catherine H. Wu\",\"doi\":\"10.1163/156855800744548\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Gene transfer to the liver by receptor-mediated endocytosis represents a promising method for therapeutic intervention of genetic disorders and acquired diseases affecting the liver. Based on the observation that asialoglycoprotein receptors are abundantly expressed on the surface of human hepatocytes in vivo, gene delivery and expression by DNA-ligand complexes targeted to liver have been developed in several different experimental models. This review will cover general aspects related to targeting to the liver via the asialoglycoprotein receptor, and the relationship between gene expression, persistence, the structure and size of DNA complexes, as well as the current strategies aimed to improve the overall efficiency of receptor-mediated gene therapy.\",\"PeriodicalId\":93646,\"journal\":{\"name\":\"Gene therapy and regulation\",\"volume\":\"1 1\",\"pages\":\"79-93\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2000-03-07\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://sci-hub-pdf.com/10.1163/156855800744548\",\"citationCount\":\"4\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Gene therapy and regulation\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1163/156855800744548\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Gene therapy and regulation","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1163/156855800744548","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Liver-selective nucleic acid targeting using the asialoglycoprotein receptor
Gene transfer to the liver by receptor-mediated endocytosis represents a promising method for therapeutic intervention of genetic disorders and acquired diseases affecting the liver. Based on the observation that asialoglycoprotein receptors are abundantly expressed on the surface of human hepatocytes in vivo, gene delivery and expression by DNA-ligand complexes targeted to liver have been developed in several different experimental models. This review will cover general aspects related to targeting to the liver via the asialoglycoprotein receptor, and the relationship between gene expression, persistence, the structure and size of DNA complexes, as well as the current strategies aimed to improve the overall efficiency of receptor-mediated gene therapy.
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