内耳精准给药赋能先天性耳聋基因治疗:策略、挑战与展望
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复旦大学附属眼耳鼻喉科医院耳鼻喉科

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Targeted Inner Ear Drug Delivery Empowering Gene Therapy for Congenital Hearing Loss: Strategies, Challenges, and Prospects
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ENT Institute and Department of Otorhinolaryngology,Eye ENT Hospital,Fudan University

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    摘要:

    随着多项针对先天性耳聋的临床试验结果公布,基因治疗有望成为根治耳聋的新型治疗策略。然而,治疗体系能否高效、精准地递送至内耳,仍是决定疗效的关键之一。本文基于内耳独特解剖与生理特征,系统梳理了内耳精准给药策略从基础研究向临床转化的发展过程,重点分析不同内耳递送路径对治疗安全性和有效性的影响。同时,结合多项针对OTOF相关耳聋的基因治疗临床试验,展示了内耳精准给药策略在临床转化研究中的实用价值,并提出新形势下内耳外科的崛起。本综述为内耳精准给药提供了系统的认识和了解,使相关从业人员具有清晰的可参考依据,有助于促进先天性耳聋基因治疗的发展和推广。特别的是,内耳外科概念的提出必将引领耳科学新的范式变革。

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    Recent clinical trials targeting congenital hearing loss have demonstrated the promise of gene therapy as a curative approach. However, whether the therapeutic 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 review systematically summarizes 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.

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  • 收稿日期:2025-12-09
  • 最后修改日期:2025-12-09
  • 录用日期:2026-01-05
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