遗传性听力障碍的基因治疗研究进展  

Recent Development of Gene Therapy for Hereditary Deafness

作　　者：令娜娜 郭玉芬[1] 徐百成[1] LING Na-na;GUO Yu-fen;XU Bai-cheng

机构地区：[1]兰州大学第二医院耳鼻咽喉头颈外科,兰州730000 [2]西安市人民医院(西安市第四医院)耳鼻咽喉头颈外科,西安710000

出　　处：《中国听力语言康复科学杂志》2023年第4期394-398,共5页Chinese Scientific Journal of Hearing and Speech Rehabilitation

基　　金：国家自然科学基金项目(31960132);甘肃省卫生行业计划项目(GSWSKY2018-40)。

摘　　要：听力障碍是常见的感觉障碍性疾病之一,目前用于治疗遗传性听力障碍的临床选择有限,主要是通过配戴助听器或植入人工耳蜗等补偿或重建听力,并不能恢复自然听力。从病理学来说,恢复生理听觉是听力障碍治疗的必然目标,因此基因治疗才是听力障碍康复的最终方法。目前遗传性听力障碍的基因治疗还处在动物实验阶段,如何强化该基础研究并促进其向临床转化,是未来研究的重点。本文对遗传性听力障碍基因治疗的现状、发展前景和挑战等进行文献复习和发展展望。Deafness is one of the common sensory disorders.At present,the clinical options for the treatment of hereditary deafness are still very limited,mainly by wearing hearing aids or undergoing cochlear implant surgery to compensate the hearing.In fact,natural hearing cannot be restored.From the pathological point of view,restoring physiological hearing is the inevitable goal of deafness treatment,so in this sense,a gene therapy is the ultimate method of deafness rehabilitation.At present,the gene therapy of hereditary deafness is still in the stage of animal experiments.How to strengthen this basic research and promote its transformation into clinical are the focus of future research.This article reviews the current status,development prospects and challenges of gene therapy for hereditary deafness.

关 键 词：遗传性听力障碍 基因治疗 靶细胞 基因载体 载体导入途径 基因置换 基因沉默 基因编辑 

分 类 号：R764.43[医药卫生—耳鼻咽喉科] R450[医药卫生—临床医学]