{"title":"肝脏疾病的基因治疗:腺相关病毒载体(AAV)和病毒样颗粒(VLPS)","authors":"Mayra V. Quinga, Lizbeth X. Quiroz","doi":"10.21931/rb/cs/2019.02.01.20","DOIUrl":null,"url":null,"abstract":"The liver has a critical role in several genetic inherited and acquired disorders. Over the years, the development of several therapies to treat liver diseases resulted in several successful treatment outcomes for liver disorders. However, its use has been severely hampering by many undesirable side effects and methodological restrictions. Currently, there are several advances for the treatment of hepatic diseases with genetic therapy, which address several problems. Research on recent new treatments has focused on the development of specific gene editing approaches that use novel genetic tools, as well as the efficient distribution systems of these tools in the liver. This paper will provide an overview of current and emerging therapeutic strategies such as Adeno-associated Virus Vectors (AAV), new serotypes of AVV for gene therapy and Virus-like particles (VLPs)","PeriodicalId":214615,"journal":{"name":"Humboldt kolleg Ibarra 2019","volume":"1 1","pages":"0"},"PeriodicalIF":0.0000,"publicationDate":"2019-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Gene therapy in liver diseases focus on Adeno-Associate Virus Vector (AAV) and Virus-Like Particles (VLPS)\",\"authors\":\"Mayra V. Quinga, Lizbeth X. Quiroz\",\"doi\":\"10.21931/rb/cs/2019.02.01.20\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"The liver has a critical role in several genetic inherited and acquired disorders. Over the years, the development of several therapies to treat liver diseases resulted in several successful treatment outcomes for liver disorders. However, its use has been severely hampering by many undesirable side effects and methodological restrictions. Currently, there are several advances for the treatment of hepatic diseases with genetic therapy, which address several problems. Research on recent new treatments has focused on the development of specific gene editing approaches that use novel genetic tools, as well as the efficient distribution systems of these tools in the liver. This paper will provide an overview of current and emerging therapeutic strategies such as Adeno-associated Virus Vectors (AAV), new serotypes of AVV for gene therapy and Virus-like particles (VLPs)\",\"PeriodicalId\":214615,\"journal\":{\"name\":\"Humboldt kolleg Ibarra 2019\",\"volume\":\"1 1\",\"pages\":\"0\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2019-02-19\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Humboldt kolleg Ibarra 2019\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.21931/rb/cs/2019.02.01.20\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Humboldt kolleg Ibarra 2019","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.21931/rb/cs/2019.02.01.20","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Gene therapy in liver diseases focus on Adeno-Associate Virus Vector (AAV) and Virus-Like Particles (VLPS)
The liver has a critical role in several genetic inherited and acquired disorders. Over the years, the development of several therapies to treat liver diseases resulted in several successful treatment outcomes for liver disorders. However, its use has been severely hampering by many undesirable side effects and methodological restrictions. Currently, there are several advances for the treatment of hepatic diseases with genetic therapy, which address several problems. Research on recent new treatments has focused on the development of specific gene editing approaches that use novel genetic tools, as well as the efficient distribution systems of these tools in the liver. This paper will provide an overview of current and emerging therapeutic strategies such as Adeno-associated Virus Vectors (AAV), new serotypes of AVV for gene therapy and Virus-like particles (VLPs)