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Articles by Xiaoting Cheng
Re-administration of AAV-mediated gene therapy for OTOF -related deafness: a single-arm trial
Abstract Re-administration of adeno-associated virus (AAV)-mediated gene therapy remains challenging due to neutralizing antibodies (NAbs) induced by the initial dose. We previously conducted a single-arm trial showing that single-dose administration of AAV-hOTOF gene therapy in individuals with OTOF-related deafness is safe and leads to hearing improvements.
Multicentre gene therapy for OTOF -related deafness followed up to 2.5 years
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Abstract Autosomal recessive deafness 9, caused by OTOF gene mutations, is characterized by severe-to-complete congenital deafness1. Although gene therapy has shown benefits in a small number of patients2,3,4,5, its safety and efficacy across broader age ranges and longer follow-up periods, as well as predictors of treatment outcomes, remain unclear.
By Xiaoting Cheng, Jun Lv, Yuxin Chen, Xiaoyun Chen, Rongqun Zhai, Liqin Zhang, Yiling Zhang, Juhong Zhang, Di Deng, Zhicheng Huang, Qi Cao, Xin Zhang, Daqi Wang, Yizhe Wang, Liheng Chen, Sha Yu Verified, Luo Guo, Bowen Zhang, Hui Wang, Yi Zhou, Liling Dai, Wei Wang, Longlong Zhang, Yanbo Yin, Guiqing Cheng, Ziyi Zhou, Wuqing Wang, Bing Chen, Wei Lu, Zhiqiang Gao, Dazhi Shi, Yuanping Xiong, Yu Zhao, Wei Yuan, Qin Wang, Guodong Feng, Huawei Li, Zheng-Yi Chen, Yilai Shu, Luoying Jiang, Hongqun Jiang
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Nature
Verified
Gene Therapy vs Cochlear Implantation in Restoring Hearing Function and Speech Perception for Individuals With Congenital Deafness
Gene Therapy vs Cochlear Implantation in Restoring Hearing Function and Speech Perception for Individuals With Congenital Deafness Supplement 2. eTable 1. Demographic and Clinical Characteristics of Each CI Patient eTable 2. Demographic and Clinical Characteristics of Each GT Patient eTable 3. Data Collection for Each CI Patient eTable 4. Data Collection for Each GT Patient eTable 5A. Comparison of Auditory Thresholds for GT-only and Matched CI Patients Using Mann Whitney U test eTable 5B.
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
Summary Autosomal recessive deafness 9, caused by mutations of the OTOF gene, is characterised by congenital or prelingual, severe-to-complete, bilateral hearing loss. However, no pharmacological treatment is currently available for congenital deafness. In this Article, we report the safety and efficacy of gene therapy with an adeno-associated virus (AAV) serotype 1 carrying a human OTOF transgene (AAV1-hOTOF) as a treatment for children with autosomal recessive deafness 9.
AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial
State Key Laboratory of Digital Medical Engineering, Southeast University, Nanjing, China Department of Otolaryngology Head and Neck Surgery of Zhongda Hospital, Southeast University, Nanjing, China Advanced Institute for Life and Health, Southeast University, Nanjing, China Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, China Co-Innovation Center of Neuroregeneration, Nantong University, Nantong, China Department of Neurology of Aerospace...
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