RNA base editing therapy cures hearing loss induced by OTOF gene mutation

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...

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Veröffentlicht in:	Molecular therapy 2023-12, Vol.31 (12), p.3520-3530
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Deafness Gene Editing Hearing Loss - genetics Hearing Loss - therapy Hearing Loss, Central Mice Mutation Original
Online-Zugang:	Volltext
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