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, doi:10.1016/S0140-6736(23)02874-X, 2024

2.Xiaoting Cheng#, Kevin A Peng, Bing Chen, Yilai Shu*.A teenager with auricular infection secondary to piercing.Bmj-British Medical Journal, 380, 2023

3.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

4.Wang H, Xun M, Tang H, Zhao J, Hu S, Zhang L, Lv J, Wang D, Chen Y, Liu J, Li GL, Wang W*, Shu Y*, Li H*. Hair cell-specific Myo15 promoter-mediated gene therapy rescues hearing in DFNB9 mouse model.Mol Ther Nucleic Acids. 2024 Feb 2;35(1):102135

5.Hu Shaowei, Lv Jun, Wang Zijing….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, 2024, Accepted

6.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

7.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

8.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

9.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

10.You Zheng#, Heng Liu#, Yuxin Chen#, Shaowei Dong#, Fang Wang, Shengyi Wang, Geng-Lin Li, Yilai Shu*, Fei Xu*. Structural insights into the lipid and ligand regulation of a human neuronal KCNQ channel. Neuron, 110(2): 237-247, 2022

11.Luoying Jiang#, Daqi Wang#, Yingzi He*, Yilai Shu*. Advances in gene therapy hold promise for treating hereditary hearing loss. Molecular Therapy, 31(4): 934-950, 2023

12.Chong Cui#, Daqi Wang#, Bowei Huang#, Fang Wang#, Yuxin Chen, Jun Lv, Luping Zhang, Lei Han, Dong Liu, Zheng-Yi Chen, Geng-Lin Li, Huawei Li, Yilai Shu*. Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss. Molecular Therapy-Nucleic Acids, 29: 400-412, 2022

13.Qing Li#*, Chong Cui#, Rongyu Liao, Xidi Yin, Daqi Wang, Yanbo Cheng, Bowei Huang, Liqin Wang, Meng Yan, Jinan Zhou, Jingjing Zhao, Wei Tang, Yingyi Wang, Xiaohan Wang, Jun Lv, Jinsong Li, Huawei Li*, Yilai Shu*. The pathogenesis of common Gjb2 mutations associated with human hereditary deafness in mice. Cellular and Molecular Life Sciences, 80(6), 2023

14.Jiali Wang#, Chong Wang, Qiao Wang, Zhuohao Zhang, Hui Wang, Shengyi Wang, Zhangcai Chi, Luoran Shang*, Wuqing Wang*, Yilai Shu*. Microfluidic Preparation of Gelatin Methacryloyl Microgels as Local Drug Delivery Vehicles for Hearing Loss Therapy. Acs Applied Materials & Interfaces, 2022

15.Pengchao Feng#, Zhijiao Xu#, Jialin Chen, Meizhen Liu, Yu Zhao, Daqi Wang, Lei Han, Li Wang, Bo Wan, Xingshun Xu, Dali Li, Yilai Shu*, Yimin Hua*. Rescue of mis-splicing of a common SLC26A4 mutant associated with sensorineural hearing loss by antisense oligonucleotides. Molecular Therapy-Nucleic Acids,  28: 280-292, 2022

16.Yang Guo#, Lei Han#, Shuang Han, Honghai Tang, Shengyi Wang, Chong Cui, Bing Chen*, Huawei Li*, Yilai Shu*. Specific knockdown of Htra2 by CRISPR-CasRx prevents acquired sensorineural hearing loss in mice. Molecular Therapy-Nucleic Acids, 28: 643-655, 2022

17.Xinde Hu#, Jinghan Wang#, Xuan Yao#, Qingquan Xiao#, Yuanyuan Xue, Shaoran Wang, Linyu Shi, Yilai Shu*, Huawei Li*, Hui Yang*. Screened AAV variants permit efficient transduction access to supporting cells and hair cells. Cell Discovery, 5, 2019

18.Xi Gu#, Daqi Wang#, Zhijiao Xu#, Jinghan Wang, Luo Guo, Renjie Chai, Genglin Li, Yilai Shu*, Huawei Li*. Prevention of acquired sensorineural hearing loss in mice by in vivo Htra2 gene editing. Genome Biology, 22(1), 2021

19.Yilai Shu#, Wenyan Li#, Mingqian Huang#, Yi-Zhou Quan#, Deborah Scheffer, Chunjie Tian, Yong Tao, Xuezhong Liu, Konrad Hochedlinger, Artur A. Indzhykulian, Zhengmin Wang, Huawei Li, Zheng-Yi Chen*. Renewed proliferation in adult mouse cochlea and regeneration of hair cells. Nature Communications, 10, 2019

20.Honghai Tang#, Hui Wang#, Shengyi Wang#, Shao Wei Hu#, Jun Lv, Mengzhao Xun, Kaiyu Gao, Fang Wang, Yuxin Chen, Daqi Wang, Wuqing Wang*, Huawei Li*, Yilai Shu*. Hearing of Otof-deficient mice restored by trans-splicing of N- and C-terminal otoferlin. Human Genetics, 142(2): 289-304, 2023

21.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*. Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates.  Molecular Therapy Methods & Clinical Development, 31, 2023

22.Shuang Han#, Zhijiao Xu#, Shengyi Wang, Honghai Tang, Shaowei Hu, Hui Wang, Guofang Guan*, Yilai Shu*. Distributional comparison of different AAV vectors after unilateral cochlear administration. Gene therapy, 2023

23.Honghai Tang#, Daqi Wang, Yilai Shu*. Structural insights into Cas9 mismatch: promising for development of high-fidelity Cas9 variants. Signal Transduction and Targeted Therapy, 7(1). 2022

24.Jinghan Wang#, Liping Zhao#, Xi Gu#, Yuanyuan Xue, Shengyi Wang, Ru Xiao, Luk H Vandenberghe, Kevin A Peng, Yilai Shu*, Huawei Li*. Efficient delivery of adeno-associated virus (AAV) into inner ear in vivo via trans-stapes route in adult guinea pig. Human Gene Therapy, 33(13-14):719-728, 2022 (Cover story) 

25.Kun Zhang#, Xiaoting Cheng#, Liping Zhao#, Mingqian Huang, Yong Tao, Hongbo Zhang, Jessica M. Rosenholm, Min Zhuang, Zheng-Yi Chen, Bing Chen*, Yilai Shu*. Direct Functional Protein Delivery with a Peptide into Neonatal and Adult Mammalian Inner Ear In Vivo. Molecular Therapy-Methods & Clinical Development, 18: 511-519. 2020

26.Bing Wang#, Tianwen Xin#, Lang Shen, Kun Zhang, Dan Zhang, Hui Zhang, Jisheng Liu, Bing Chen*, Wenguo Cui*, Yilai Shu*. Acoustic transmitted electrospun fibrous membranes for tympanic membrane regeneration. Chemical Engineering Journal, 419, 2021

27.Xiaoting Cheng#, Jialin Liu, Ning Li, Eric Nisenbaum, Qing Sun, Bing Chen, Roy Casiano, Donald Weed, Fred Telischi, James C. Denneny, Iii, Xuezhong Liu*, Yilai Shu*. Otolaryngology Providers Must Be Alert for Patients with Mild and Asymptomatic COVID-19. Otolaryngology-Head and Neck Surgery, 162(6): 809-810. 2020

28.Kun Zhang#, Yanqing Fang#, Bing Wang, Xiaoting Cheng, Bing Chen*, Yilai Shu*. Frequency-specific hearing results after stapes surgery for Chinese population otosclerosis with different degrees of hearing loss. Acta Otolaryngol, 140(5): 356-360. 2020

29.Yilai Shu#, Lijun Wang#, Xiaoting Cheng, Chayada Tangshewinsirikul, Weili Shi, Yasheng Yuan, Zhiqiang Yang, Huawei Li, Jun Shen*, Bing Chen*, Weiguo Zou*. The p.Pro170Leu Variant in NOG Impairs Noggin Secretion and Causes Autosomal Dominant Congenital Conductive Hearing Loss Due to Stapes Ankylosis. Journal of Genetics and Genomics, 46(9):445-449, 2019

30.Jinghan Wang#, Jun Shen#, Luo Guo, Cheng Cheng, Renjie Chai, Yilai Shu* Huawei Li*. A humanized mouse model demonstrated that apparently autosomal dominant deafness caused by MYO6 p.C442Y is inherited in a semi-dominant pattern. Hearing research, 379:79-88, 2019

31.Yilai Shu#, Yong Tao, Zhengmin Wang, Yong Tang, Huawei Li, Pu Dai, Guangping Gao, Zheng-Yi Chen*. Identification of Adeno-associated viral vectors (AAV) that target neonatal and adult mammalian inner ear cell subtypes. Human Gene Therapy, 27(9):687-99, 2016(Cover story)

32.John A Zuris#, David B Thompson, Yilai Shu, John P Guilinger, Jeffrey L Bessen, Johnny H Hu, Morgan L Maeder, J Keith Joung, Zheng-Yi Chen, David R Liu*. Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo. Nature Biotechnology, 33(1):73-80, 2015

33.Xiaoting Cheng#, Yangwenyi Liu#, Yilai Shu*, Duo-Duo Tao, Bing Wang, Yasheng Yuan, John J. Galvin, Iii, Qian-Jie Fu, Bing Chen*. Music Training Can Improve Music and Speech Perception in Pediatric Mandarin-Speaking Cochlear Implant Users. Trends in Hearing, 22, 2018

34.Xue Gao#, Yong Tao#, Veronica Lamas, Mingqian Huang, Wei-Hsi Yeh, Bifeng Pan, Yu-Juan Hu, Johnny H Hu, David B Thompson, Yilai Shu, Yamin Li, Hongyang Wang, Shiming Yang, Qiaobing Xu, Daniel B Polley, M Charles Liberman, Wei-Jia Kong, Jeffrey R Holt, Zheng-Yi Chen*, David R Liu*. Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents. Nature, 553(7687):217-221, 2018