AAV‐Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness

AAV‐Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness

Abstract Mutations in OTOFERLIN (OTOF) lead to the autosomal recessive deafness 9 (DFNB9). The efficacy of adeno‐associated virus (AAV)‐mediated OTOF gene replacement therapy is extensively validated in Otof‐deficient mice. However, the clinical safety and efficacy of AAV‐OTOF is not reported. Here,...

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Bibliographic Details
Main Authors: Jieyu Qi, Fangzhi Tan, Liyan Zhang, Ling Lu, Shanzhong Zhang, Yabo Zhai, Yicheng Lu, Xiaoyun Qian, WenXiu Dong, Yinyi Zhou, Ziyu Zhang, Xuehan Yang, Lulu Jiang, Chaorong Yu, Jiancheng Liu, Tian Chen, Lianqiu Wu, Chang Tan, Sijie Sun, Huaien Song, Yilai Shu, Lei Xu, Xia Gao, Huawei Li, Renjie Chai
Format: Article
Language:English
Published: Wiley 2024-03-01
Series:Advanced Science
Subjects:
AAV
biosafety
clinical trial
hearing recovery
OTOF gene therapy
Online Access:https://doi.org/10.1002/advs.202306788

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