Article
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial.
Lancet (London, England) - 25 May 2024
Lv Jun, Wang Hui, Cheng Xiaoting, Chen Yuxin, Wang Daqi, Zhang Longlong, Cao Qi, Tang Honghai, Hu Shaowei, Gao Kaiyu, Xun Mengzhao, Wang Jinghan, Wang Zijing, Zhu Biyun, Cui Chong, Gao Ziwen, Guo Luo, Yu Sha, Jiang Luoying, Yin Yanbo, Zhang Jiajia, Chen Bing, Wang Wuqing, Chai Renjie, Chen Zheng-Yi, Li Huawei, Shu Yilai
Abstract excerpt
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 efficacy of gene therapy with an adeno-associated virus (AAV) serotype 1 carrying a human OTOF transgene...
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