Advancements in gene therapy for Huntington's disease
裴中 1吴腾腾2
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作者信息
1. 中山大学附属第一医院神经科,广州 510080
2. 广州医科大学附属第一医院神经内科,广州 510080
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摘要
亨廷顿病是一种常染色体显性遗传病,近年来多项针对mRNA水平的干预策略相继开展临床试验,同时随着聚集的规律性间隔短回文重复序列(clustered regularly interspersed short palindromic repeats,CRISPR)/CRISPR 关联基因(CRISPR asso-ciated gene,Cas)系统的日渐成熟,针对致病基因组的基因编辑策略也屡有报道.本文将围绕亨廷顿病基因治疗的临床现状、研究进展、临床评估的改进做简要综述.
Abstract
Huntington's disease is an autosomal dominant genetic disease.In recent years,clinical trials have been conducted for vari-ous intervention strategies targeting mRNA levels,and meanwhile,with the development of the clustered regularly interspersed short palindromic repeat(CRISPR)/CRISPR-associated genes system,there are also reports on gene editing strategies for pathogenic ge-nomes.This article reviews the gene therapy for Huntington's disease in terms of current clinical status,research advances,and im-provements in clinical assessment.
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