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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Detalles Bibliográficos
Autores principales: Zhang, Longlong, Wang, Hui, Xun, Mengzhao, Tang, Honghai, Wang, Jinghan, Lv, Jun, Zhu, Biyun, Chen, Yuxin, Wang, Daqi, Hu, Shaowei, Gao, Ziwen, Liu, Jianping, Chen, Zheng-Yi, Chen, Bing, Li, Huawei, Shu, Yilai
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10679773/
https://www.ncbi.nlm.nih.gov/pubmed/38027066
http://dx.doi.org/10.1016/j.omtm.2023.101154

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