在美国,一名三岁患有亨特综合症的男孩成为英国境外第一个接受基因疗法治疗的男孩,这种疗法阻止了他的病情发展并恢复了关键功能。 A three-year-old boy with Hunter syndrome in the U.S. became the first outside the UK to receive a gene therapy that halted his disease’s progression and restored key functions.

一位三岁男孩患有Hunter综合症(一种罕见的遗传病),成为世界上第一个在联合王国境外接受突破性基因治疗的人,在2025年2月一次性治疗后,情况显著改善。 A three-year-old boy with Hunter syndrome, a rare genetic disorder, has become the first in the world to receive a groundbreaking gene therapy outside the UK, showing remarkable improvements after a one-time treatment in February 2025. 在曼彻斯特开发的治疗 使用改良干细胞 提供功能性基因 使身体产生一种缺失的酶 包括大脑中的酶 抑制神经衰减 The therapy, developed in Manchester, uses modified stem cells to deliver a functional gene that enables the body to produce a missing enzyme, including in the brain, halting neurological decline. 自从治疗以来,男孩Oliver Chu获得了流动性、言语和认知能力,不再需要每周的酶注射。 Since treatment, the boy, Oliver Chu, has gained mobility, speech, and cognitive skills, and no longer needs weekly enzyme infusions. 测试证实酶水平是正常的数百倍 Tests confirm enzyme levels are now hundreds of times normal. 实验性治疗是涉及5名男孩的一项试验的一部分,它为具有类似遗传条件的儿童带来了希望,尽管它仍在长期研究之中,而且尚未广泛提供。 The experimental treatment, part of a trial involving five boys, offers hope for children with similar genetic conditions, though it remains under long-term study and is not yet widely available.