Progress in Gene Therapy of Hereditary Neurological Ion Channel Disease

Xiaoyi Wang, Wen-So Su, Tiankuo Xue, Yu-lan Zhu
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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
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遗传性神经离子通道病基因治疗研究进展
目前遗传性神经病变的治疗方法往往难以控制或患者耐受性低,因此探索新的治疗方法势在必行。基因治疗在许多研究中已被证明可以应用于神经系统疾病,并可能在未来成为遗传性神经系统离子通道疾病的有效治疗方法。同时,光遗传学、化学遗传学和基因编辑技术的不断进步也为遗传性神经病基因治疗的发展提供了良好的基础。本文将对遗传性神经离子通道病的基因治疗方法、机制、转导载体、启动子和给药途径进行综述。关键词:离子通道病;基因治疗;基因治疗;基因编辑;CRISPR / Cas9
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来源期刊
CiteScore
0.10
自引率
0.00%
发文量
16855
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.
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