Valentini Chris, Szeto Betsy, Kysar Jeffrey W, Lalwani Anil K
Department of Otolaryngology -- Head and Neck Surgery, Columbia University Vagelos College of Physicians and Surgeons, New York, NY.
Department of Mechanical Engineering, School of Engineering, Columbia University, New York, New York.
Hearing Balance Commun. 2020;18(4):278-285. doi: 10.1080/21695717.2020.1807261. Epub 2020 Aug 25.
Current treatments for hearing loss offer some functional improvements in hearing, but do not restore normal hearing. The aim of this review is to highlight recent advances in viral and non-viral vectors for gene therapy and to discuss approaches for overcoming barriers inherent to inner ear delivery of gene products.
The databases used were Medline, EMBASE, Web of Science, and Google Scholar. Search terms were [("cochlea*" or "inner ear" or "transtympanic" or "intratympanic" or "intracochlear" or "hair cells" or "spiral ganglia" or "Organ of Corti") and ("gene therapy" or "gene delivery")]. The references section of resulting articles was also used to identify relevant studies.
Both viral and non-viral vectors play important roles in advancing gene delivery to the inner ear. The round window membrane is one significant barrier to gene delivery that intratympanic delivery methods attempt to overcome through diffusion and intracochlear delivery methods bypass completely.
Gene therapy for hearing loss is a promising treatment for restoring hearing function by addressing innate defects. Recent technological advances in inner ear drug delivery techniques pose exciting opportunities for progress in gene therapy.
目前针对听力损失的治疗方法在听力功能方面有一定改善，但无法恢复正常听力。本综述旨在强调基因治疗中病毒载体和非病毒载体的最新进展，并讨论克服基因产物内耳递送固有障碍的方法。
使用的数据库有Medline、EMBASE、科学网和谷歌学术。检索词为[（“耳蜗*”或“内耳”或“经鼓膜”或“鼓室内”或“蜗内”或“毛细胞”或“螺旋神经节”或“柯蒂氏器”）以及（“基因治疗”或“基因递送”）]。所得文章的参考文献部分也用于识别相关研究。
病毒载体和非病毒载体在推进基因递送至内耳方面都发挥着重要作用。圆窗膜是基因递送的一个重要障碍，鼓室内递送方法试图通过扩散来克服这一障碍，而蜗内递送方法则完全绕过该障碍。
通过解决先天性缺陷，听力损失的基因治疗是恢复听力功能的一种有前景的治疗方法。内耳药物递送技术的最新技术进展为基因治疗的进步带来了令人兴奋的机遇。
