摘要
超过60%的语前聋病例是由于遗传因素引起。遗传性耳聋患者的基因突变包括单核苷酸替换、碱基缺失、插入等,当这些突变导致耳聋基因的错义时便可临床上引起遗传性耳聋的症状。对这些突变在基因水平选择性纠正是目前遗传性耳聋生物治疗的新思路。CRISpR/Cas技术作为第三代的靶向基因组编辑技术系统,具有靶向、高效、易操作等优势,很大程度上推进了耳聋的基因治疗研究。本文对目前国内外耳聋的基因治疗研究现状进行回顾。
More than 60%of the Prelingual deafness cases are caused by genetic factors.Mutations in hereditary deafness include single nucleotide substitution,base deletion and insertion.These mutations lead to missense and nonsense mutations in the deafness gene.Selective correction at gene level is an innovative way to treat hereditary deafness.CRISPR/Cas technology has the advantages of targeting,high efficiency and easy operation as the third generation of targeted genome editing technology.It greatly promotes the research of gene therapy for hearing loss.In this paper,the current research status of gene therapy for hearing loss was reviewed.
作者
顾湘
郭维维
杨仕明
GU Xiang;GUO Wei-wei;YANG Shi-ming
出处
《中国听力语言康复科学杂志》
2019年第4期275-279,共5页
Chinese Scientific Journal of Hearing and Speech Rehabilitation
基金
国家自然科学基金面上项目(81670940,81670941,81570933)
北京市生命科学优势项目-干细胞联合人工耳蜗植入治疗耳聋的探索性研究
北京科技创新基地培育与发展专项(z151100001615050)
