Department of Molecular and Cellular Neuroscience, Dorris Neuroscience Center, The Scripps Research Institute, 10550 North Torrey Pines Road, La Jolla, San Diego, California 92037, USA.
Oregon Hearing Research Center, Vollum Institute, Oregon Health &Science University, 3181 South West Sam Jackson Park Road, Portland, Oregon 97239, USA.
Nat Rev Drug Discov. 2015 May;14(5):346-65. doi: 10.1038/nrd4533. Epub 2015 Mar 20.
Hearing loss is the most common form of sensory impairment in humans and affects more than 40 million people in the United States alone. No drug-based therapy has been approved by the Food and Drug Administration, and treatment mostly relies on devices such as hearing aids and cochlear implants. Over recent years, more than 100 genetic loci have been linked to hearing loss and many of the affected genes have been identified. This understanding of the genetic pathways that regulate auditory function has revealed new targets for pharmacological treatment of the disease. Moreover, approaches that are based on stem cells and gene therapy, which may have the potential to restore or maintain auditory function, are beginning to emerge.
听力损失是人类最常见的感觉障碍形式,仅在美国就影响了超过 4000 万人。食品和药物管理局尚未批准任何基于药物的治疗方法,治疗主要依赖于助听器和人工耳蜗等设备。近年来,已有 100 多个遗传位点与听力损失相关,许多受影响的基因已被确定。对调节听觉功能的遗传途径的这种理解为该疾病的药物治疗提供了新的靶点。此外,基于干细胞和基因治疗的方法,可能具有恢复或维持听觉功能的潜力,正在开始出现。