掌握耳鼻咽喉头颈外科疾病诊治，擅长耳科手术及耳聋三级防控。聚焦耳聋基因治疗研发及临床转化，研发出OTOF耳聋基因治疗药物和装备，主导全球首个先天性耳聋基因治疗临床试验，纠正聋哑患者的听力、言语和声源定位能力，实现耳聋的病因学根治和颠覆性范式转变。以通讯作者发表SCI论文60余篇，包括The Lancet、BMJ、Nature Medicine、JAMA Neurology、Nature Biomedical Engineering、Nature Human Behaviour等著名期刊。牵头首个遗传性耳聋基因治疗国际专家共识。获美国ARO临床科学创新奖（亚洲首位）、上海市青年科技杰出贡献奖、科学探索奖等。主持包括国自然创新研究群体、科技部重点研发课题、国家杰青、优青、面上、上海市科委科技创新行动计划细胞与基因治疗专项等多项纵向课题。培养团队多名成员获上海市超级博士后、国自然科学基金、“晨光计划”等。
He has mastered the diagnosis and treatment of diseases in otorhinolaryngology-head and neck surgery, and is proficient in otologic surgery and the three-level prevention and control of deafness. He focuses on the research, development, and clinical translation of gene therapy for deafness, has developed the OTOF deafness gene therapy drug and equipment, and led the world's first clinical trial of gene therapy for congenital deafness, which corrected deaf-mute patients' hearing, speech, and sound localization abilities, achieving an etiological cure and a disruptive paradigm shift for deafness. As a corresponding author, he has published over 60 SCI papers, including in renowned journals such as The Lancet, Nature Medicine, JAMA Neurology, and Nature Biomedical Engineering. He led the development of the first international expert consensus on gene therapy for hereditary deafness. He has received awards including the ARO Award for Clinical Innovation (the first in Asia), Shanghai Youth Outstanding Science and Technology Contribution Award, and the Xplorer Prize. He has presided over multiple vertical projects including the National Key R&D Program of China, National Natural Science Foundation of China for National Science Fund for Innovative Research Group, Distinguished Young Scholars, Excellent Young Scientists Fund Program, General Program, and Shanghai Municipal Commission of Science and Technology's Science and Technology Innovation Action Plan for Cell and Gene Therapy. Trained several team members who received grants including the Shanghai Post-doctoral Excellence Program, the National Natural Science Foundation of China (NSFC) grants, and the Shanghai "Morning Light Plan" project.
【代表性论文/Representative Publications】
1.Jun Lv#, Hui Wang#, Xiaoting Cheng#, Yuxin Chen#, Daqi Wang#, Longlong Zhang, Qi Cao, Honghai Tang, Shaowei Hu, Kaiyu Gao, Mengzhao Xun, Jinghan Wang, Zijing Wang, Biyun Zhu, Chong Cui, Ziwen Gao, Luo Guo, Sha Yu, Luoying Jiang, Yanbo Yin, Jiajia Zhang, Bing Chen, Wuqing Wang*, Renjie Chai*, Zheng-Yi Chen*, Huawei Li*, and Yilai Shu*. AAV1-hOTOF Gene Therapy for Autosomal Recessive Deafness 9: a single-arm trial. The Lancet, 403(10441):2317-2325, 2024. (Cover story)
2.Hui Wang#, Yuxin Chen#, Jun Lv#, Xiaoting Cheng#, Qi Cao#, Daqi Wang, Longlong Zhang, Biyun Zhu, Min Shen, Chunxin Xu, Mengzhao Xun, Zijing Wang, Honghai Tang, Shaowei Hu, Chong Cui, Luoying Jiang, Yanbo Yin, Luo Guo, Yi Zhou, Lei Han, Ziwen Gao, Jiajia Zhang, Sha Yu, Kaiyu Gao, Jinghan Wang, Bing Chen, Wuqing Wang*, Zheng-Yi Chen*, Huawei Li*, Yilai Shu*. Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results. Nature Medicine,30(7):1898-1904, 2024.
3.Xiaoting Cheng#, Kevin A Peng, Bing Chen, Yilai Shu*. A teenager with auricular infection secondary to piercing. The British Medical Journal, 380:e071715, 2023.
4.Chong Cui#, Shengyi Wang#, Daqi Wang#, Jingjing Zhao, Bowei Huang, Biyun Zhu, Yuxin Chen, Honghai Tang, Yu Han, Cheng Ye, Dan Mu, Chengdong Zhang, Yuan Yang, Yihan Bao, Jun Lv, Shuang Han, Geng-Lin Li, Huawei Li, Yilai Shu*. A base editor for the long-term restoration of auditory function in mice with recessive profound deafness. Nature Biomedical Engineering, 9(1):40-56, 2025.
5.Jiajia Zhang#, Zengzhi Guo#, Changjie Pan#, Chunchun Hu#, Xinyang Weng#, Yang-wenyi Liu, Xiaoting Cheng, Jun Lv, Qi Cao, Hui Wang, Yuxin Chen, Daqi Wang, Shaowei Hu, Mengzhao Xun, Longlong Zhang, Zijing Wang, Honghai Tang, Biyun Zhu, Luo Guo, Sha Yu, Xiaoling Hu, Lin Chen, Bing Chen, Zheng-Yi Chen, Shan Sun*, Xiu Xu*, Huawei Li*, Fei Chen* and Yilai Shu*. Preliminary evidence for enhanced auditory cortex activation and mental development after gene therapy in children with autosomal recessive deafness 9. Nature Human Behaviour, 9(7):1457-1469, 2025.
6.Xiaoting Cheng#, Jiake Zhong#, Jiajia Zhang#, Chong Cui#, Luoying Jiang#, Yang-wenyi Liu#, Yuxin Chen, Qi Cao, Daqi Wang, Guiqing Cheng, Yuxin Zong, Min Shen, Chunxin Xu, Jun Lv, Hui Wang, Longlong Zhang, Biyun Zhu, Honghai Tang, Jinghan Wang, Xintai Fan, Yanqing Fang, Luo Guo, Jiawei Guo, Liheng Chen, Yanbo Yin, Zijing Wang, Lei Han, Shaowei Hu, Shengyi Wang, Guoyou Qin, Xuezhong Liu, Jinqiu Sang, Fangang Zeng, Wuqing Wang*, Bing Chen*, Zheng-Yi Chen*, Huawei Li*, Yilai Shu*. Gene Therapy vs Cochlear Implantation in Restoring Hearing Function and Speech Perception for Individuals With Congenital Deafness. JAMA Neurology, 2025 July 21.
7.Yuxin Chen#, Jiake Zhong, Yilai Shu*. Gene therapy for deafness: we can do more. Nature Reviews Genetics. 26(4):225-226, 2025.
8.Qingquan Xiao#, Zhijiao Xu#, Yuanyuan Xue#, Chunlong Xu#, Lei Han, Yuanhua Liu, Fang Wang, Runze Zhang, Shuang Han, Xing Wang, Geng-Lin Li, Huawei Li*, Hui Yang*, Yilai Shu*. Rescue of autosomal dominant hearing loss by in vivo delivery of mini dCas13X-derived RNA base editor. Science Translational Medicine, 14(654):eabn0449, 2022.
9.Xi Gu#, Xinde Hu#, Daqi Wang#, Zhijiao Xu, Fang Wang, Di Li, Geng-lin Li, Hui Yang, Huawei Li*, Erwei Zuo* and Yilai Shu*. Treatment of autosomal recessive hearing loss via in vivo CRISPR/Cas9-mediated optimized homology-directed repair in mice. Cell Research, 32(7):699-702,2022.
10.Longlong Zhang#, Dingding Dong#, Yanbo Yin#, Honghai Tang#, Jun Lv, Qi Cao, Wuqing Wang, Bing Chen*, Yunfeng Wang*, Huawei Li*, Daqi Wang*, Yilai Shu*. Audiological characteristics following gene therapy in patients with autosomal recessive deafness 9. Med, 6(8):100696, 2025. (Cover story)
11.Shao Wei Hu#, Sohyang Jeong#, Luoying Jiang#, Hansol Koo#, Zijing Wang, Won Hoon Choi, Biyun Zhu, Heeyoung Seok, Yi Zhou, Min Gu Kim, Dan Mu, Huixia Guo, Ziyi Zhou, Sung Ho Jung, Yingting Zhang, Ho Byung Chae, Liheng Chen, Sung-Yeon Lee, Luo Guo, Myung-Whan Suh, Yang Xiao, Moo Kyun Park, Honghai Tang, Jae-Jin Song, Xi Chen, Ai Chen, Jun Ho Lee, Sangsu Bae*, Sang-Yeon Lee*, Yilai Shu*.PAM-flexible adenine base editing rescues hearing loss in a humanized MPZL2 mouse model harboring an East Asian founder mutation. Nature Communications, 16(1):7186, 2025.
12.Yuanyuan Xue#, Xinde Hu#, Daqi Wang#, Di Li#, Yige Li, Fang Wang, Mingqian Huang, Xi Gu, Zhijiao Xu, Jinan Zhou, Jinghan Wang, Renjie Chai, Jun Shen, Zheng-Yi Chen, Geng-Lin Li, Hui Yang, Huawei Li*, Erwei Zuo*, and Yilai Shu*. Gene editing in a Myo6 semi-dominant mouse model rescues auditory function. Molecular Therapy, 30(1):105-118, 2022.
