一个有严重听力损失的3岁女孩在实验基因治疗后获得了正常听力,标志着全球临床试验的重大突破。 A 3-year-old girl with severe hearing loss gained normal hearing after experimental gene therapy, marking a major breakthrough in a global clinical trial.

一个3岁女孩,Opal Sandy,因OTOF基因突变而严重失去听力,在2023年11个月大时接受实验性基因治疗后,已恢复正常听力。 A 3-year-old girl, Opal Sandy, born with profound hearing loss due to an OTOF gene mutation, has regained normal hearing after receiving experimental gene therapy in 2023 at 11 months old. 该治疗是以Regenenron为主导的DB-OTO基因疗法临床试验的一部分,涉及将功能性基因复制件注入她的右脑部,同时将装置植入她的左耳。 The treatment, part of a Regeneron-led clinical trial for DB-OTO gene therapy, involved injecting a functional gene copy into her right cochlea while implanting a device in her left ear. 两年后,她听得很清楚,没有依靠植入手术,这是她父母报告的一项突破。 Two years later, she hears clearly without relying on the implant, a breakthrough reported by her parents. 在12名审判参加者中,11名在数周内明显改善了听力,没有产生严重的副作用。 Among 12 trial participants, 11 showed significant hearing improvements within weeks, with no serious side effects. 审判在英国、美国、西班牙和德国继续进行,招收18岁以下的儿童。 The trial continues in the U.K., U.S., Spain, and Germany, enrolling children under 18.