中国耳鼻咽喉颅底外科杂志2025，Vol.31Issue(6)：1-10,10.DOI:10.11798/j.issn.1007-1520.202525475
内耳精准给药赋能先天性耳聋基因治疗的策略、挑战与展望
Targeted inner ear drug delivery empowering gene therapy for congenital hearing loss:strategies,challenges and prospects
摘要
Abstract
Recent clinical trials targeting congenital hearing loss have demonstrated the promise of gene therapy as a curative approach.However,whether the treatment system can deliver agents to the inner ear efficiently and precisely remains one of the key determinants of therapeutic efficacy.Based on the unique anatomical and physiological features of the inner ear,this article systematically reviews the development process of targeted inner ear drug delivery strategies from basic research to clinical translation,with a focus on analyzing the impacts of different inner ear delivery pathways on treatment safety and effectiveness.Meanwhile,drawing on multiple gene therapy clinical trials for OTOF-related deafness,it highlights the practical value of targeted inner ear delivery strategies in clinical translation and the emergence of inner ear surgery in the current landscape.This review provides a systematic perspective on targeted inner ear delivery strategies,offering a clear reference framework for relevant practitioners and facilitating the development and translation of gene therapy for congenital hearing loss.Furthermore,the introduction of the inner ear surgery concept is poised to herald a new paradigm in otology.关键词
先天性耳聋/基因治疗/内耳局部递送/腺相关病毒/内耳外科Key words
Congenital hearing loss/Gene therapy/Local delivery to the inner ear/Adeno-associated virus/Inner ear surgery分类
医药卫生引用本文复制引用
舒易来,蔡尚璇,范新泰,张龙龙,王镜涵..内耳精准给药赋能先天性耳聋基因治疗的策略、挑战与展望[J].中国耳鼻咽喉颅底外科杂志,2025,31(6):1-10,10.基金项目
国家杰出青年科学基金(82225014). （82225014）
