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...
“gene therapy”, “DFNB9 trial”, “DFNB9 mouse”, or combinations thereof. We also searchedClinicalTrials.govfor related clinical trials. We found proof-of-principle of gene therapy for autosomal recessive deafness 9 in animal models using recombinant adeno-associated viral vectors and four ...
Background Autosomal recessive deafness 9, caused by mutations of the OTOF gene, is characterised by congenital or prelingual, severe-to-complete, bilateral hearing loss . However, no pharmacological treatment is currently available for congenital deafness . In this Article, we report the safety and...
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 develop a clinical application of AAV1-hOTOF gene therapy, we evaluated its efficacy and safety in animal models using pharmacodynamics, behavior, and histopathology. AAV1-hOTOF inner ear delivery significantly improved hearing in Otof −/− mice without affecting normal hearing in wild-...
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...