{"title":"基于非经典三脚架干扰RNA结构格式的先进治疗平台","authors":"S. Sajeesh, Dong Ki Lee","doi":"10.14800/RD.708","DOIUrl":null,"url":null,"abstract":"The flexibility of human RNAi (RNA interference) machinery to accommodate unconventional structures has opened the way for the development of novel RNAi triggering formats, suitable for advanced therapeutic applications. Non-classical siRNA structural variants show improved functional features over the conventional siRNA formats including increased potency, reduced non-specific responses, and enhanced cellular delivery. These non-classical RNAi structures with enhanced structural and functional properties, in combination with appropriate delivery vehicles, can be an effective therapeutic module. Here we summarize our recent efforts on expanding the structural repertoire of classical siRNAs, using tripodal interfering RNA formats, to develop novel RNAi based therapeutics for advanced health-care applications.","PeriodicalId":90965,"journal":{"name":"RNA & disease (Houston, Tex.)","volume":"2 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2015-03-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"1","resultStr":"{\"title\":\"Advanced Therapeutic Platforms based on Non-Classical Tripodal Interfering RNA Structural Format\",\"authors\":\"S. Sajeesh, Dong Ki Lee\",\"doi\":\"10.14800/RD.708\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"The flexibility of human RNAi (RNA interference) machinery to accommodate unconventional structures has opened the way for the development of novel RNAi triggering formats, suitable for advanced therapeutic applications. Non-classical siRNA structural variants show improved functional features over the conventional siRNA formats including increased potency, reduced non-specific responses, and enhanced cellular delivery. These non-classical RNAi structures with enhanced structural and functional properties, in combination with appropriate delivery vehicles, can be an effective therapeutic module. Here we summarize our recent efforts on expanding the structural repertoire of classical siRNAs, using tripodal interfering RNA formats, to develop novel RNAi based therapeutics for advanced health-care applications.\",\"PeriodicalId\":90965,\"journal\":{\"name\":\"RNA & disease (Houston, Tex.)\",\"volume\":\"2 1\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2015-03-22\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"RNA & disease (Houston, Tex.)\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.14800/RD.708\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"RNA & disease (Houston, Tex.)","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.14800/RD.708","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Advanced Therapeutic Platforms based on Non-Classical Tripodal Interfering RNA Structural Format
The flexibility of human RNAi (RNA interference) machinery to accommodate unconventional structures has opened the way for the development of novel RNAi triggering formats, suitable for advanced therapeutic applications. Non-classical siRNA structural variants show improved functional features over the conventional siRNA formats including increased potency, reduced non-specific responses, and enhanced cellular delivery. These non-classical RNAi structures with enhanced structural and functional properties, in combination with appropriate delivery vehicles, can be an effective therapeutic module. Here we summarize our recent efforts on expanding the structural repertoire of classical siRNAs, using tripodal interfering RNA formats, to develop novel RNAi based therapeutics for advanced health-care applications.