rAAV在遗传性耳聋治疗中的研究进展  

Advances in rAAV in the Treatment of Hereditary Deafness

作　　者：魏鼎 白心悦 杜嘉妮 王卓灵 王青燕 陈雅慧[1] 翟晓燕 WEI Ding;BAI Xinyue;DU Jiani;WANG Zhuoling;WANG Qingyan;CHEN Yahui;ZHAI Xiaoyan(School of Medicine,Yan’an University,Yan’an 716000,China)

机构地区：[1]延安大学医学院,延安716000

出　　处：《中华耳科学杂志》2024年第5期861-867,共7页Chinese Journal of Otology

基　　金：国家自然科学基金项目(82360223);延安大学校级科研项目(205040404);延安大学校级大学生创新创业项目(D2022144)。

摘　　要：据世界卫生组织2021年报道,50%以上先天性听力损失是由遗传性因素变异引起的。临床上用于提高耳聋患者听力的几种主要方式(如佩戴助听器、植入人工耳蜗)都有一定局限性。近年来,重组腺相关病毒(recombination adeno-associated virus,r AAV)作为相对安全的基因治疗载体在遗传性疾病的临床及基础研究中被广泛应用。单r AAV由于包装载体大小受限,在大基因片段缺陷造成的疾病研究与治疗中的应用受限。目前已有团队开发了双重r AAV用于治疗大基因片段缺陷造成的疾病。文章着重介绍了双重r AAV在大基因片段缺陷造成的耳聋中的研究进展,为将来开发更多耳聋基因治疗r AAV提供帮助。According to the World Health Organization 2021,more than 50%of congenital hearing loss is caused by genetic variants of factors.Several of the main clinical modalities used to improve hearing in hearing loss patients(e.g.,hearing aids,cochlear implants)have limitations.Recombination adeno-associated virus(rAAV)has been widely used as a relatively safe gene therapy vector in clinical and basic research of genetic diseases.The application of single rAAV in the research and treatment of diseases caused by defects in large gene fragments is limited due to the restricted size of the packaging vector.Currently,teams have developed dual rAAVs for the treatment of diseases caused by defects in large gene fragments.This review highlights the progress of dual rAAV in deafness diseases caused by defective large gene fragments to help develop more gene therapy rAAV for deafness diseases in the future.

关 键 词：遗传性耳聋 重组腺相关病毒 双重重组腺相关病毒 基因治疗 

分 类 号：R764.43[医药卫生—耳鼻咽喉科]