成功的病毒介导基因治疗后，首批DFNB9患者听觉脑干反应逐渐恢复。

IF 11.8 Q1 MEDICINE, RESEARCH & EXPERIMENTAL

Med Pub Date : 2025-08-08 DOI:10.1016/j.medj.2025.100775

Barbara Vona, Nicola Strenzke

{"title":"成功的病毒介导基因治疗后，首批DFNB9患者听觉脑干反应逐渐恢复。","authors":"Barbara Vona, Nicola Strenzke","doi":"10.1016/j.medj.2025.100775","DOIUrl":null,"url":null,"abstract":"A bit more than one year ago, there was a major breakthrough in the treatment of sensorineural hearing loss: three publications1,2,3 reported on the first successful gene therapy trials to restore hearing function in children with autosomal recessive prelingual deafness DFNB9. In their recent publication in Med, Zhang et al.4 report follow-up data regarding objective measures of hearing function.","PeriodicalId":29964,"journal":{"name":"Med","volume":"6 8","pages":"100775"},"PeriodicalIF":11.8000,"publicationDate":"2025-08-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Gradual recovery of auditory brainstem responses in the first DFNB9 patients with successful virus-mediated gene therapy.\",\"authors\":\"Barbara Vona, Nicola Strenzke\",\"doi\":\"10.1016/j.medj.2025.100775\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"A bit more than one year ago, there was a major breakthrough in the treatment of sensorineural hearing loss: three publications1,2,3 reported on the first successful gene therapy trials to restore hearing function in children with autosomal recessive prelingual deafness DFNB9. In their recent publication in Med, Zhang et al.4 report follow-up data regarding objective measures of hearing function.\",\"PeriodicalId\":29964,\"journal\":{\"name\":\"Med\",\"volume\":\"6 8\",\"pages\":\"100775\"},\"PeriodicalIF\":11.8000,\"publicationDate\":\"2025-08-08\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Med\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1016/j.medj.2025.100775\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"MEDICINE, RESEARCH & EXPERIMENTAL\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Med","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/j.medj.2025.100775","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, RESEARCH & EXPERIMENTAL","Score":null,"Total":0}

引用次数: 0

摘要

一年多以前，在治疗感音神经性听力损失方面取得了重大突破：三篇出版物1,2,3报道了首次成功的基因治疗试验，以恢复常染色体隐性语前耳聋DFNB9儿童的听力功能。Zhang等人最近在《Med》杂志上发表了关于听力功能客观测量的随访数据。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Gradual recovery of auditory brainstem responses in the first DFNB9 patients with successful virus-mediated gene therapy.

A bit more than one year ago, there was a major breakthrough in the treatment of sensorineural hearing loss: three publications¹^,²^,³ reported on the first successful gene therapy trials to restore hearing function in children with autosomal recessive prelingual deafness DFNB9. In their recent publication in Med, Zhang et al.⁴ report follow-up data regarding objective measures of hearing function.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Med MEDICINE, RESEARCH & EXPERIMENTAL-

CiteScore

17.70

自引率

0.60%

发文量

102

期刊介绍： Med is a flagship medical journal published monthly by Cell Press, the global publisher of trusted and authoritative science journals including Cell, Cancer Cell, and Cell Reports Medicine. Our mission is to advance clinical research and practice by providing a communication forum for the publication of clinical trial results, innovative observations from longitudinal cohorts, and pioneering discoveries about disease mechanisms. The journal also encourages thought-leadership discussions among biomedical researchers, physicians, and other health scientists and stakeholders. Our goal is to improve health worldwide sustainably and ethically. Med publishes rigorously vetted original research and cutting-edge review and perspective articles on critical health issues globally and regionally. Our research section covers clinical case reports, first-in-human studies, large-scale clinical trials, population-based studies, as well as translational research work with the potential to change the course of medical research and improve clinical practice.