Department of Otolaryngology-Head and Neck Surgery, Columbia University Medical Center, New York Presbyterian Hospital, New York, New York 10032.
Department of Otolaryngology-Head and Neck Surgery, University of California San Francisco, San Francisco, California 94117.
Cold Spring Harb Perspect Med. 2019 Sep 3;9(9):a033191. doi: 10.1101/cshperspect.a033191.
Over 450 million people worldwide suffer from hearing loss, leading to an estimated economic burden of ∼$750 billion. The past decade has seen significant advances in the understanding of the molecular mechanisms that contribute to hearing, and the environmental and genetic factors that can go awry and lead to hearing loss. This in turn has sparked enormous interest in developing gene therapy approaches to treat this disorder. This review documents the most recent advances in cochlear gene therapy to restore hearing loss, and will cover viral vectors and construct designs, potential routes of delivery into the inner ear, and, lastly, the most promising genes of interest.
全球有超过 4.5 亿人患有听力损失，估计造成的经济负担约为 7500 亿美元。在过去的十年中，人们对导致听力损失的分子机制以及可能出错的环境和遗传因素有了更深入的了解。这反过来又极大地激发了人们对开发基因治疗方法来治疗这种疾病的兴趣。本文回顾了耳蜗基因治疗恢复听力损失的最新进展，将涵盖病毒载体和构建设计、内耳递药的潜在途径，以及最后最有前途的感兴趣基因。
