Chien Wade W, Monzack Elyssa L, McDougald Devin S, Cunningham Lisa L
1Section on Sensory Cell Biology, National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, Maryland, USA; and 2Department of Otolaryngology-Head and Neck Surgery, Johns Hopkins School of Medicine, Baltimore, Maryland, USA.
Ear Hear. 2015 Jan;36(1):1-7. doi: 10.1097/AUD.0000000000000088.
Gene therapy is a promising treatment modality that is being explored for several inherited disorders. Multiple human gene therapy clinical trials are currently ongoing, but few are directed at hearing loss. Hearing loss is one of the most prevalent sensory disabilities in the world, and genetics play an important role in the pathophysiology of hearing loss. Gene therapy offers the possibility of restoring hearing by overcoming the functional deficits created by the underlying genetic mutations. In addition, gene therapy could potentially be used to induce hair cell regeneration by delivering genes that are critical to hair cell differentiation into the cochlea. In this review, we examine the promises and challenges of applying gene therapy to the cochlea. We also summarize recent studies that have applied gene therapy to animal models of hearing loss.
基因治疗是一种很有前景的治疗方式，目前正在针对多种遗传性疾病进行探索。目前正在进行多项人类基因治疗临床试验，但针对听力损失的试验很少。听力损失是世界上最普遍的感官残疾之一，遗传学在听力损失的病理生理学中起着重要作用。基因治疗提供了通过克服潜在基因突变造成的功能缺陷来恢复听力的可能性。此外，基因治疗有可能通过将对毛细胞分化至关重要的基因导入耳蜗来诱导毛细胞再生。在这篇综述中，我们探讨了将基因治疗应用于耳蜗的前景和挑战。我们还总结了最近将基因治疗应用于听力损失动物模型的研究。
