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SHU Yilai

Name:
SHU Yilai
Position:
Vice President of Eye & ENT Hospital of Fudan University and the director of the Diagnosis and Treatment Center for Genetic Hearing Loss, Director of Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases (Preparation)
Title:
M.D., Ph.D., Professor, Physician Scientist
TEL:
（021） 6437 7134  
Email:
   yilai_shu@fudan.edu.cn
Research Interests:

1) Health and Chronic Disease Management                  

2) Interdisciplinary Nursing                  

3) Maternal-Child Nursing                  

BIOGRAPHY

Yilai Shu is a professor and physician-scientist in the Department of Otolaryngology at the Eye & ENT Hospital of Fudan University. He serves as the associate dean of the Institute of Otorhinolaryngology and the director of the Diagnosis and Treatment Center for Hereditary Deafness. Prof. Shu is renowned for his expertise in gene therapy for hereditary deafness, dedicating his career to unraveling the pathogenic mechanism of deafness and pioneering innovative therapeutic strategies, including gene replacement and CRISPR/Cas9 gene editing. He also actively promotes clinical translation to benefit patients. He and collaborators developed AAV1-hOTOF, a gene therapy drug designed to treat DFNB9, a form of deafness caused by mutations in the OTOF gene. He led the first-in-human clinical trial to investigate the safety and efficacy of gene therapy for hereditary deafness. The results were remarkable, demonstrating that AAV1-hOTOF could effectively restore auditory function and speech perception in patients. These findings were published in prestigious journals such as The Lancet and Nature Medicine, marking a significant milestone in the field. His contributions have been recognized through numerous awards, including the National Science Fund for Distinguished Young Scholars of the National Natural Science Foundation of China, Shanghai Youth Outstanding Science and Technology Contribution Award，Shanghai Municipal Hospital Clinical Innovation Award，the Silver Snake Award of Shanghai, the Shanghai Youth May Fourth Medal, and the Travel Award of the Association for Research in Otolaryngology of USA, etc.

EDUCATION & TRAINING

  • Oct 2010 - Jun 2012

    Joint training program student between Harvard Medical School and Fudan University

  • Sep 2007 - Jun 2012

    M.D. and Ph.D. Degree in Otolaryngology, Fudan University

  • Sep 1999 - Jul 2004

    Bachelor's Degree in Medicine, University of South China

TEACHING & RESEARCH

Teaching Areas:

  Otolaryngology - Head & Neck Surgery (Ear, Nose and Throat);
Evidence-based Nursing, Nursing Research

Research Grants:

  1. Ongoing Grants as PI：

    1. National Natural Science Foundation of China (NSFC),   ￥4,000,000, 202301-202712.

    2. the National Key R&D Program of China,   ￥4,580,000, 202011-202510.

    3. the Science and Technology Commission of Shanghai Municipality, ￥3,000,000, 202312-202611.the National Key R&D Program of China,   ￥4,580,000, 202011-202510.

    4. National Natural Science Foundation of China (NSFC),  ￥570,000, 202201-202512.

  2.    Finished Grants as PI：

    1. National Natural Science Foundation of China (NSFC),  ￥1,300,000, 201901-202112.

    2. National Natural Science Foundation of China (NSFC),  ￥560,000, 201801-202112.

    3. National Natural Science Foundation of China (NSFC),  ￥230,000, 201401-201612.

    4. the Science and Technology Commission of Shanghai Municipality, ￥500,000, 201804-202103

    5. the Science and Technology Commission of Shanghai Municipality, ￥200,000, 201705-202004

    6. the Science and Technology Commission of Shanghai Municipality, ￥200,000, 201507-201706

ACADEMIC SERVICE

  • 2023-Present Committee member, Chinese Society of Otolaryngology-Head and Neck Surgery；

  • 2023-Present Chairperson of the Expert Committee for Auditory Medicine Innovation and Translation of Audiology Development Foundation of China；

  • 2023-Present Committee member, Shanghai Medical Association Otolaryngology-Head and Neck Surgery Specialty Branch；

  • 2023-Present Committee member, Shanghai Medical Association Rare Diseases Specialty Branch；

  • 2021-2025 Standing committee member, Hearing, Speech and Communication Studies Academic Subgroup of the Biophysical Society of China；

  • 2018-2021 Committee member, Long Range Planning of ARO；

  • 2015-2018 Committee member, International Committee of ARO；

SELECTED 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, 2024. DOI:10.1038/s 41591-024-03023-5. Epub 2024 Jun 5.

  3. Xiaoting Cheng#, Kevin A Peng, Bing Chen, Yilai Shu*. A teenager with auricular infection secondary to piercing. Bmj-British Medical Journal, 2023 Mar 16;380:e071715. doi: 10.1136/bmj-2022-071715.

  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, 2024. DOI:10.1038/s41551-024-01235-1.

  5. Shao Wei Hu#, Jun Lv#, Zijing Wang#, Honghai Tang#, Hui Wang, Fang Wang, Daqi Wang, Juan Zhang, Longlong Zhang, Qi Cao, Yuxin Chen, Ziwen Gao, Yu Han, Wuqing Wang, Geng-Lin Li*, Yilai Shu*, Huawei Li*. Engineering of the AAV-Compatible Hair Cell-Specific Small-Size Myo15 Promoter for Gene Therapy in the Inner Ear. Research, 7, 2024. DOI:10.34133/research.0341.

  6. 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

  7. 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

  8. 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

  9. Ziwen Zheng#, Guo Li#, Chong Cui#, Fang Wang, Xiaohan Wang, Zhijiao Xu, Huiping Guo, Yuxin Chen, Honghai Tang, Daqi Wang, Mingqian Huang, Zheng-Yi Chen, Xingxu Huang, Huawei Li, Geng-Lin Li*, Xiaoxiang Hu* and Yilai Shu*. Preventing autosomal-dominant hearing loss in Bth mice with CRISPR/CasRx-based RNA editing. Signal Transduction and Targeted Therapy, 7(1):79, 2022

  10.    Qianying Liu#, Xiang Zhang#, Hui Huang#, Yuxin Chen#, Fang Wang, Aihua Hao, Wuqiang Zhan, Qiyu Mao, Yuxia Hu, Lin Han, Yifang Sun, Meng Zhang, Zhimin Liu, Geng-Lin Li, Weijia Zhang, Yilai Shu*, Lei Sun*, Zhenguo Chen*. Asymmetric pendrin homodimer reveals its molecular mechanism as anion exchanger. Nature Communications, 14(1), 2023

Updated On:2025-04-14