遗传性神经系统离子通道病基因治疗的研究进展

Progress in Gene Therapy of Hereditary Neurological Ion Channel Disease

目的遗传性神经系统离子通道病现有的治疗方法往往难以控制病情或者患者耐受度较低,因此探索新的治疗方法势在必行。基因治疗在多项研究中显示可能应用于神经系统疾病,并可能成为未来遗传性神经系统离子通道病的有效治疗手段。同时,光遗传学、化学遗传学方法、基因编辑技术的不断进步也为遗传性神经系统离子通道病的基因治疗提供了良好的发展基础。我们将就遗传性神经系统离子通道病基因治疗的方法机制、转导载体、启动子及给药途径作一综述。

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.