Article
RNA base editing therapy cures hearing loss induced by OTOF gene mutation.
Molecular therapy : the journal of the American Society of Gene Therapy - 6 Dec 2023
Xue Yuanyuan, Tao Yong, Wang Xing, Wang Xueling, Shu Yilai, Liu Yuanhua, Kang Wen, Chen Sifan, Cheng Zhenzhe, Yan Boou, Xie Yanwei, Bi Lanting, Jia Haitao, Li Jinhui, Xiao Qingquan, Chen Liying, Yao Xuan, Shi Linyu, Yang Hui, Wu Hao
Abstract excerpt
Otoferlin (OTOF) gene mutations represent the primary cause of hearing impairment and deafness in auditory neuropathy. The c.2485C>T (p. Q829X) mutation variant is responsible for approximately 3% of recessive prelingual deafness cases within the Spanish population. Previous studies have used two recombinant AAV vectors to overexpress OTOF, albeit with limited efficacy. In this study, we introduce an enhanced...
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