人类遗传性听力损失的潜在治疗方法：当前的难题。
Potential treatments for genetic hearing loss in humans: current conundrums.
机构信息
Departments of Otolaryngology-Head and Neck Surgery, Kumamoto University, Graduate School of Medicine, Kumamoto City, Kumamoto, Japan.
出版信息
Gene Ther. 2015 Aug;22(8):603-9. doi: 10.1038/gt.2015.27. Epub 2015 Mar 17.
Genetic defects are a major cause of hearing loss in newborns. Consequently, hearing loss has a profound negative impact on human daily living. Numerous causative genes for genetic hearing loss have been identified. However, presently, there are no truly curative treatments for this condition. There have been several recent reports on successful treatments in mice using embryonic gene therapy, neonatal gene therapy and neonatal antisense oligonucleotide therapy. Herein, we describe state-of-the-art research on genetic hearing loss treatment through gene therapy and discuss the obstacles to overcome in curative treatments of genetic hearing loss in humans.
遗传性缺陷是导致新生儿耳聋的主要原因。因此，耳聋对人类的日常生活有深远的负面影响。已经确定了许多遗传性听力损失的致病基因。然而，目前这种疾病没有真正有效的治疗方法。最近有几篇关于在小鼠中使用胚胎基因治疗、新生儿基因治疗和新生儿反义寡核苷酸治疗成功治疗的报道。本文通过基因治疗描述了遗传性听力损失治疗的最新研究进展，并讨论了人类遗传性听力损失治疗中需要克服的障碍。
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