Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates

Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates

Pathogenic mutations in the OTOF gene cause autosomal recessive hearing loss (DFNB9), one of the most common forms of auditory neuropathy. There is no biological treatment for DFNB9. Here, we designed an OTOF gene therapy agent by dual-adeno-associated virus 1 (AAV1) carrying human OTOF coding seque...

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Bibliographic Details
Main Authors: Longlong Zhang, Hui Wang, Mengzhao Xun, Honghai Tang, Jinghan Wang, Jun Lv, Biyun Zhu, Yuxin Chen, Daqi Wang, Shaowei Hu, Ziwen Gao, Jianping Liu, Zheng-Yi Chen, Bing Chen, Huawei Li, Yilai Shu
Format: Article
Language:English
Published: Elsevier 2023-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hereditary hearing loss
gene therapy
dual-AAV delivery
DFNB9
AAV1-hOTOF
efficacy and safety
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001936

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