{"title":"Progress in Gene Therapy of Hereditary Neurological Ion Channel Disease","authors":"Xiaoyi Wang, Wen-So Su, Tiankuo Xue, Yu-lan Zhu","doi":"10.3760/CMA.J.ISSN.1008-6315.2019.04.020","DOIUrl":null,"url":null,"abstract":"Current therapies for hereditary neuropathy are often difficult to control or have low patient tolerance, so it is imperative to explore new therapies.Gene therapy has been shown that in many studies it may be applied to nervous system diseases and may become an effective treatment for hereditary nervous system ion channel diseases in the future.At the same time, the continuous progress of photogenetics, chemical genetics and gene editing technology also provides a good basis for the development of gene therapy for hereditary neuropathy.We will review the methods, mechanisms, transduction vectors, promoters and administration routes of gene therapy for hereditary neurological ion channel disease. \n \n \nKey words: \nIon channel disease; Gene therapy; Gene therapy; Gene editing; CRISPR/Cas9","PeriodicalId":10365,"journal":{"name":"中国综合临床","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2019-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"中国综合临床","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3760/CMA.J.ISSN.1008-6315.2019.04.020","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
Abstract
Current therapies for hereditary neuropathy are often difficult to control or have low patient tolerance, so it is imperative to explore new therapies.Gene therapy has been shown that in many studies it may be applied to nervous system diseases and may become an effective treatment for hereditary nervous system ion channel diseases in the future.At the same time, the continuous progress of photogenetics, chemical genetics and gene editing technology also provides a good basis for the development of gene therapy for hereditary neuropathy.We will review the methods, mechanisms, transduction vectors, promoters and administration routes of gene therapy for hereditary neurological ion channel disease.
Key words:
Ion channel disease; Gene therapy; Gene therapy; Gene editing; CRISPR/Cas9
期刊介绍:
Clinical Medicine of China is an academic journal organized by the Chinese Medical Association (CMA), which mainly publishes original research papers, reviews and commentaries in the field.
Clinical Medicine of China is a source journal of Peking University (2000 and 2004 editions), a core journal of Chinese science and technology, an academic journal of RCCSE China Core (Extended Edition), and has been published in Chemical Abstracts of the United States (CA), Abstracts Journal of Russia (AJ), Chinese Core Journals (Selection) Database, Chinese Science and Technology Materials Directory, Wanfang Database, China Academic Journal Database, JST Japan Science and Technology Agency Database (Japanese) (2018) and other databases.