肝脏疾病的基因治疗:腺相关病毒载体(AAV)和病毒样颗粒(VLPS)

Mayra V. Quinga, Lizbeth X. Quiroz
{"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}
引用次数: 0

摘要

肝脏在一些遗传、遗传和获得性疾病中起着关键作用。多年来,几种治疗肝脏疾病的疗法的发展导致了肝脏疾病的几种成功治疗结果。然而,它的使用受到许多不良副作用和方法限制的严重阻碍。目前,在肝脏疾病的基因治疗方面有一些进展,解决了几个问题。最近对新疗法的研究主要集中在开发使用新型遗传工具的特定基因编辑方法,以及这些工具在肝脏中的有效分配系统。本文将概述当前和新兴的治疗策略,如腺相关病毒载体(AAV),用于基因治疗的新型AVV血清型和病毒样颗粒(vlp)。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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)
求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
自引率
0.00%
发文量
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信