6月5日Nature Medicine报道的研究“Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results”显示,单臂的腺相关病毒1型(AAV1)携带人类OTOF基因的基因治疗对患有常染色体隐性耳聋9型(DFNB9)...
6月5日 Nature Medicine报道的研究“Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results”显示,单臂的腺相关病毒1型(AAV1)携带人类OTOF基因的基因治疗对患有常染色体隐性耳聋9型(DFNB9)的患者是安全的,并且在功能上有一定的改善效果。随后,研究方案得到了修订和批准,...
6月5日 Nature Medicine报道的研究“Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results”显示,单臂的腺相关病毒1型(AAV1)携带人类OTOF基因的基因治疗对患有常染色体隐性耳聋9型(DFNB9)的患者是安全的,并且在功能上有一定的改善效果。随后,研究方案得到了修订和批准,...
6月5日 Nature Medicine报道的研究“Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results”显示,单臂的腺相关病毒1型(AAV1)携带人类OTOF基因的基因治疗对患有常染色体隐性耳聋9型(DFNB9)的患者是安全的,并且在功能上有一定的改善效果。随后,研究方案得到了修订和批准,...
近日,复旦大学舒易来等多团队合作在国际知名医学期刊Lancet(IF=169)在线发表题为“AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial”的研究论文,这项单臂、单中心试验招募了患有严重至完全听力损失、证实有OTOF两个等位基因突变、未植入双侧耳蜗的儿童(1-18岁)。AAV1-hOTOF单...
AAV1-hOTOF gene therapy is safe and efficacious as a novel treatment for children with autosomal recessive deafness 9. Funding National Natural Science Foundation of China, National Key R&D Program of China, Science and Technology Commission of Shanghai Municipality, and Shanghai Refreshgene Therapeu...
Findings Between Oct 19, 2022, and June 9, 2023, we screened 425 participants for eligibility and enrolled six children for AAV1-hOTOF gene therapy (one received a dose of 9×1011 vector genomes [vg] and five received 1·5×1012 vg). All participants completed follow-up visits up to ...
③The Lancet:AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial基于腺相关病毒(AAV)的AAV1-hOTOF用于常染色体隐性遗传性耳聋9型(DFNB9)的一项单臂试验DOI: 10.1016/S0140-6736(23)02874-X④JAMA:Protein Score May Reclassify Death Risk in Patients With Heart Failure蛋白质...
To evaluate the relevance of our gene therapy approach for humans, we also injected AAV1-hOTOF into adult Otof−/− mice at P30 with different doses via the RWM and then tested their auditory function. At 2 weeks after injection, the click ABR thresholds in the injected ear of the ...
登上《柳叶刀》:复旦大学舒易来等多团队发布国际首个耳聋基因治疗临床数据,成功恢复患者听力。2024年1月25日,复旦大学附属眼耳鼻喉科医院舒易来、李华伟、王武庆,哈佛大学医学院陈正一、东南大学柴人杰等在国际顶尖医学期刊《柳叶刀》(The Lancet)上发表了题为:AAV1-hOTOF gene therapy for autosomal recessive deafness...