腺相关病毒介导的基因治疗在预防获得性听力损失中的最新进展。
Current Advances in Adeno-Associated Virus-Mediated Gene Therapy to Prevent Acquired Hearing Loss.
机构信息
Department of Pathology and Laboratory Medicine, Medical University of South Carolina, Walton Research Building, Room 403-E, 39 Sabin Street, Charleston, SC, 29425, USA.
Department of Otolaryngology, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, China.
出版信息
J Assoc Res Otolaryngol. 2022 Oct;23(5):569-578. doi: 10.1007/s10162-022-00866-y. Epub 2022 Aug 24.
Adeno-associated viruses (AAVs) are viral vectors that offer an excellent platform for gene therapy due to their safety profile, persistent gene expression in non-dividing cells, target cell specificity, lack of pathogenicity, and low immunogenicity. Recently, gene therapy for genetic hearing loss with AAV transduction has shown promise in animal models. However, AAV transduction for gene silencing or expression to prevent or manage acquired hearing loss is limited. This review provides an overview of AAV as a leading gene delivery vector for treating genetic hearing loss in animal models. We highlight the advantages and shortcomings of AAV for investigating the mechanisms and preventing acquired hearing loss. We predict that AAV-mediated gene manipulation will be able to prevent acquired hearing loss.
腺相关病毒 (AAV) 是一种病毒载体，由于其安全性、在非分裂细胞中的持续基因表达、靶向细胞特异性、无致病性和低免疫原性，为基因治疗提供了极好的平台。最近，使用 AAV 转导进行基因治疗遗传性听力损失在动物模型中显示出前景。然而，AAV 转导用于基因沉默或表达以预防或管理获得性听力损失的情况有限。本综述提供了 AAV 作为治疗动物模型遗传性听力损失的主要基因传递载体的概述。我们强调了 AAV 的优缺点，用于研究机制和预防获得性听力损失。我们预测，AAV 介导的基因操作将能够预防获得性听力损失。
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