International expert consensus on gene therapy for hereditary hearing loss: Based on clinical trials

Xintai Fan¹, Ziwen Gao¹, Jiake Zhong², Yuxin Chen¹, Xiaoyun Chen², Lukas D Landegger³, Tobias Moser⁴, Fan-Gang Zeng⁵, Yu Sun⁶, Xin Jin⁷, Robert Nash⁸, Wade W Chien⁹, Dan Jiang¹⁰, John H Greinwald¹¹, Manohar Bance¹², Manuel Manrique Rodríguez¹³, Sang-Yeon Lee¹⁴, Guodong Feng¹⁵, Haidi Yang¹⁶, Chen-Chi Wu¹⁷, Lei Xu¹⁸, Wei Yuan¹⁹, Yong Feng²⁰, Yu Zhao²¹, Barbara Vona²², Nicola Strenzke²³, Dirk Beutner²⁴, Nikul Amin²⁵, James Arwyn-Jones²⁶, Deepak Chandrasekeharan²⁵, Dazhi Shi²⁷, Di Zhang²⁸, Jianming Yang²⁹, Jieyu Qi³⁰, Qin Wang³¹, Yanbo Yin³², Yen-Fu Cheng³³, Yong Tao³⁴, Yongfu Yu³⁵, Daqi Wang¹, Luoying Jiang², Luo Guo¹, Liheng Chen¹, Xiaoting Cheng¹, Chong Cui¹, Jun Lv¹, Shuang Han¹, Wuqing Wang³², Yongxin Li³⁶, Xia Gao³⁷, Xue Zhong Liu³⁸, Dingjun Zha³⁹, Haibo Shi⁴⁰, Bing Chen³², Qiuju Wang⁴¹, Huijun Yuan⁴², Shiming Yang⁴³, Shankai Yin⁴⁰, Hao Wu⁴⁴, Zhengmin Wang¹, Huawei Li², Jay T Rubinstein⁴⁵, Lawrence R Lustig⁴⁶, Renjie Chai⁴⁷, Zheng-Yi Chen⁴⁸, Yilai Shu⁴⁹

Affiliations

¹ ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China.
² ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China; State Key Laboratory of Brain Function and Disorders and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China; Institutes of Biomedical Sciences, Fudan University, Shanghai, China.
³ Department of Otolaryngology-Head and Neck Surgery, Stanford University School of Medicine, Palo Alto, CA, USA; Department of Otolaryngology-Head and Neck Surgery, Medical University of Vienna, Vienna, Austria.
⁴ Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany; Cluster of Excellence "Multiscale Bioimaging: from Molecular Machines to Networks of Excitable Cells" (MBExC), University of Göttingen, Göttingen, Germany; Department of Otolaryngology, University Medical Center Göttingen, Göttingen, Germany; Auditory Neuroscience and Synaptic Nanophysiology Group, Max Planck Institute for Multidisciplinary Sciences, Göttingen, Germany.
⁵ Center for Hearing Research, Departments of Anatomy and Neurobiology, Biomedical Engineering, Cognitive Sciences, Otolaryngology-Head and Neck Surgery, University of California, Irvine, Irvine, CA, USA.
⁶ Department of Otolaryngology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China.
⁷ Chinese Journal of Otorhinolaryngology Head and Neck Surgery, Beijing, China.
⁸ Department of Paediatric Otolaryngology, Great Ormond Street Hospital, London, UK; University College London, Great Ormond Street Institute of Child Health, London, UK.
⁹ Department of Otolaryngology-Head and Neck Surgery, Johns Hopkins School of Medicine, Baltimore, MD, USA.
¹⁰ Hearing Implant Centre, St. Thomas Hospital, Guy's and St. Thomas' NHS Foundation Trust, London, UK; Centre for Craniofacial and Regenerative Biology, King's College London, London, UK.
¹¹ Division of Otolaryngology, Cincinnati Children's Hospital Medical Center, Department of Otolaryngology, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
¹² Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK.
¹³ Departamento de Otorrinolaringología, Clínica Universidad de Navarra, Pamplona, Navarra, Spain.
¹⁴ Department of Otorhinolaryngology-Head and Neck Surgery, Department of Genomic Medicine, Precision Medicine & Rare Disease Center, Seoul National University Hospital & Seoul National University College of Medicine, Seoul, Korea.
¹⁵ Department of Otorhinolaryngology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
¹⁶ Department of Otolaryngology, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China.
¹⁷ Department of Otolaryngology, Taiwan University Hospital, Taipei, Taiwan, China.
¹⁸ Department of Otolaryngology-Head and Neck Surgery, Shandong Provincial ENT Hospital, Shandong University, Jinan, Shandong, China.
¹⁹ Department of Otolaryngology, Chongqing General Hospital, Chongqing, China.
²⁰ Department of Otorhinolaryngology, the Affiliated Changsha Central Hospital, Hengyang Medical School, University of South China, Changsha, Hunan, China.
²¹ Department of Otolaryngology Head and Neck Surgery, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
²² Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany; Cluster of Excellence "Multiscale Bioimaging: from Molecular Machines to Networks of Excitable Cells" (MBExC), University of Göttingen, Göttingen, Germany; Institute of Human Genetics, University Medical Center Göttingen, Göttingen, Germany.
²³ Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany; Department of Otolaryngology, University Medical Center Göttingen, Göttingen, Germany.
²⁴ Department of Otolaryngology, University Medical Center Göttingen, Göttingen, Germany.
²⁵ Great Ormond Street Hospital, London, UK.
²⁶ University College London, Great Ormond Street Institute of Child Health, London, UK.
²⁷ Department of Otorhinolaryngology, the Second Affiliated Hospital, Hengyang Medical School, University of South China, Hengyang, Hunan, China.
²⁸ Department of Otolaryngology, Shenzhen Third People's Hospital, Shenzhen, China.
²⁹ Department of Otolaryngology-Head and Neck Surgery, The Second Affiliated Hospital of Anhui Medical University, Hefei, Anhui, China.
³⁰ Department of Neurology, Aerospace Center Hospital, School of Life Science, Beijing Institute of Technology, Beijing, China; State Key Laboratory of Hearing and Balance Science, Beijing Institute of Technology, Beijing, China; School of Medical Engineering, Affiliated Zhuhai People's Hospital, Beijing Institute of Technology, Zhuhai, Guangdong, China; Advanced Technology Research Institute, Beijing Institute of Technology, Jinan, Shandong, China.
³¹ Department of Otolaryngology and Head and Neck Surgery, the Second Xiangya Hospital, Central South University, Changsha, Hunan, China.
³² ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China.
³³ Department of Otolaryngology-Head and Neck Surgery, Taipei Veterans General Hospital, Taiwan, China; Institute of Brain Science, Yang Ming Chiao Tung University, Taiwan, China.
³⁴ Department of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China; Ear Institute, Shanghai Jiao Tong University School of Medicine, Shanghai, China; Shanghai Key Laboratory of Translational Medicine on Ear and Nose Diseases, Shanghai, China.
³⁵ School of Public Health and the Key Laboratory of Public Health Safety of Ministry of Education, Fudan University, Shanghai, China.
³⁶ Department of Otorhinolaryngology, Beijing Tongren Hospital, Capital Medical University, Beijing, China.
³⁷ Department of Otolaryngology-Head and Neck Surgery, Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School, Nanjing, Jiangsu, China.
³⁸ Department of Otolaryngology, University of Miami Miller School of Medicine, Miami, FL, USA.
³⁹ Department of Otorhinolaryngology Head and Neck Surgery, Xijing Hospital of Air Force Medical University, Xi'an, Shaanxi, China.
⁴⁰ Shanghai Key Laboratory of Sleep Disordered Breathing, Department of Otolaryngology-Head and Neck Surgery, Otolaryngology Institute of Shanghai Jiao Tong University, Shanghai Sixth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.
⁴¹ Department of Audiology and Vestibular Medicine, Senior Department of Otolaryngology Head and Neck Surgery, The Sixth Medical Center of PLA General Hospital, Chinese PLA Medical School, Medical Building, Beijing, China.
⁴² Institute of Rare Diseases, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
⁴³ State Key Laboratory of Hearing and Balance Science, Beijing Institute of Technology, Beijing, China; Senior Department of Otolaryngology Head and Neck Surgery, Chinese PLA General Hospital, Chinese PLA Medical School, Beijing, China; National Clinical Research Center for Otolaryngologic Diseases, Beijing, China; Key Laboratory of Hearing Science, Ministry of Education, Beijing, China; Beijing Key Laboratory of Hearing Impairment Prevention and Treatment, Beijing, China.
⁴⁴ Department of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
⁴⁵ Virginia Merrill Bloedel Hearing Research Center, Department of Otolaryngology-Head and Neck Surgery, University of Washington, Seattle, WA, USA.
⁴⁶ Department of Otolaryngology - Head and Neck Surgery, NewYork-Presbyterian Hospital/Columbia University Medical Center, New York, NY, USA. Electronic address: lrl2125@cumc.columbia.edu.
⁴⁷ Department of Neurology, Aerospace Center Hospital, School of Life Science, Beijing Institute of Technology, Beijing, China; State Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, Jiangsu, China; Co-Innovation Center of Neuroregeneration, Nantong University, Nantong, Jiangsu, China; Department of Otolaryngology Head and Neck Surgery, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, Sichuan, China; Southeast University Shenzhen Research Institute, Shenzhen, Guangdong, China. Electronic address: renjiec@seu.edu.cn.
⁴⁸ Eaton-Peabody Laboratories, Department of Otolaryngology-Head and Neck Surgery, Mass Eye & Ear/Harvard Medical School, Boston, MA, USA. Electronic address: zheng-yi_chen@meei.harvard.edu.
⁴⁹ ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China; State Key Laboratory of Brain Function and Disorders and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China; Institutes of Biomedical Sciences, Fudan University, Shanghai, China. Electronic address: yilai_shu@fudan.edu.cn.

PMID: 41135524
DOI: 10.1016/j.medj.2025.100886

Free article

International expert consensus on gene therapy for hereditary hearing loss: Based on clinical trials

Xintai Fan et al. Med. 2025.

Free article

. 2025 Oct 23:100886.

doi: 10.1016/j.medj.2025.100886. Online ahead of print.

Authors

Affiliations

¹ ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China.
² ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China; State Key Laboratory of Brain Function and Disorders and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China; Institutes of Biomedical Sciences, Fudan University, Shanghai, China.
³ Department of Otolaryngology-Head and Neck Surgery, Stanford University School of Medicine, Palo Alto, CA, USA; Department of Otolaryngology-Head and Neck Surgery, Medical University of Vienna, Vienna, Austria.
⁴ Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany; Cluster of Excellence "Multiscale Bioimaging: from Molecular Machines to Networks of Excitable Cells" (MBExC), University of Göttingen, Göttingen, Germany; Department of Otolaryngology, University Medical Center Göttingen, Göttingen, Germany; Auditory Neuroscience and Synaptic Nanophysiology Group, Max Planck Institute for Multidisciplinary Sciences, Göttingen, Germany.
⁵ Center for Hearing Research, Departments of Anatomy and Neurobiology, Biomedical Engineering, Cognitive Sciences, Otolaryngology-Head and Neck Surgery, University of California, Irvine, Irvine, CA, USA.
⁶ Department of Otolaryngology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China.
⁷ Chinese Journal of Otorhinolaryngology Head and Neck Surgery, Beijing, China.
⁸ Department of Paediatric Otolaryngology, Great Ormond Street Hospital, London, UK; University College London, Great Ormond Street Institute of Child Health, London, UK.
⁹ Department of Otolaryngology-Head and Neck Surgery, Johns Hopkins School of Medicine, Baltimore, MD, USA.
¹⁰ Hearing Implant Centre, St. Thomas Hospital, Guy's and St. Thomas' NHS Foundation Trust, London, UK; Centre for Craniofacial and Regenerative Biology, King's College London, London, UK.
¹¹ Division of Otolaryngology, Cincinnati Children's Hospital Medical Center, Department of Otolaryngology, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
¹² Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK.
¹³ Departamento de Otorrinolaringología, Clínica Universidad de Navarra, Pamplona, Navarra, Spain.
¹⁴ Department of Otorhinolaryngology-Head and Neck Surgery, Department of Genomic Medicine, Precision Medicine & Rare Disease Center, Seoul National University Hospital & Seoul National University College of Medicine, Seoul, Korea.
¹⁵ Department of Otorhinolaryngology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
¹⁶ Department of Otolaryngology, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China.
¹⁷ Department of Otolaryngology, Taiwan University Hospital, Taipei, Taiwan, China.
¹⁸ Department of Otolaryngology-Head and Neck Surgery, Shandong Provincial ENT Hospital, Shandong University, Jinan, Shandong, China.
¹⁹ Department of Otolaryngology, Chongqing General Hospital, Chongqing, China.
²⁰ Department of Otorhinolaryngology, the Affiliated Changsha Central Hospital, Hengyang Medical School, University of South China, Changsha, Hunan, China.
²¹ Department of Otolaryngology Head and Neck Surgery, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
²² Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany; Cluster of Excellence "Multiscale Bioimaging: from Molecular Machines to Networks of Excitable Cells" (MBExC), University of Göttingen, Göttingen, Germany; Institute of Human Genetics, University Medical Center Göttingen, Göttingen, Germany.
²³ Institute for Auditory Neuroscience and InnerEarLab, University Medical Center Göttingen, Göttingen, Germany; Department of Otolaryngology, University Medical Center Göttingen, Göttingen, Germany.
²⁴ Department of Otolaryngology, University Medical Center Göttingen, Göttingen, Germany.
²⁵ Great Ormond Street Hospital, London, UK.
²⁶ University College London, Great Ormond Street Institute of Child Health, London, UK.
²⁷ Department of Otorhinolaryngology, the Second Affiliated Hospital, Hengyang Medical School, University of South China, Hengyang, Hunan, China.
²⁸ Department of Otolaryngology, Shenzhen Third People's Hospital, Shenzhen, China.
²⁹ Department of Otolaryngology-Head and Neck Surgery, The Second Affiliated Hospital of Anhui Medical University, Hefei, Anhui, China.
³⁰ Department of Neurology, Aerospace Center Hospital, School of Life Science, Beijing Institute of Technology, Beijing, China; State Key Laboratory of Hearing and Balance Science, Beijing Institute of Technology, Beijing, China; School of Medical Engineering, Affiliated Zhuhai People's Hospital, Beijing Institute of Technology, Zhuhai, Guangdong, China; Advanced Technology Research Institute, Beijing Institute of Technology, Jinan, Shandong, China.
³¹ Department of Otolaryngology and Head and Neck Surgery, the Second Xiangya Hospital, Central South University, Changsha, Hunan, China.
³² ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China.
³³ Department of Otolaryngology-Head and Neck Surgery, Taipei Veterans General Hospital, Taiwan, China; Institute of Brain Science, Yang Ming Chiao Tung University, Taiwan, China.
³⁴ Department of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China; Ear Institute, Shanghai Jiao Tong University School of Medicine, Shanghai, China; Shanghai Key Laboratory of Translational Medicine on Ear and Nose Diseases, Shanghai, China.
³⁵ School of Public Health and the Key Laboratory of Public Health Safety of Ministry of Education, Fudan University, Shanghai, China.
³⁶ Department of Otorhinolaryngology, Beijing Tongren Hospital, Capital Medical University, Beijing, China.
³⁷ Department of Otolaryngology-Head and Neck Surgery, Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School, Nanjing, Jiangsu, China.
³⁸ Department of Otolaryngology, University of Miami Miller School of Medicine, Miami, FL, USA.
³⁹ Department of Otorhinolaryngology Head and Neck Surgery, Xijing Hospital of Air Force Medical University, Xi'an, Shaanxi, China.
⁴⁰ Shanghai Key Laboratory of Sleep Disordered Breathing, Department of Otolaryngology-Head and Neck Surgery, Otolaryngology Institute of Shanghai Jiao Tong University, Shanghai Sixth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.
⁴¹ Department of Audiology and Vestibular Medicine, Senior Department of Otolaryngology Head and Neck Surgery, The Sixth Medical Center of PLA General Hospital, Chinese PLA Medical School, Medical Building, Beijing, China.
⁴² Institute of Rare Diseases, West China Hospital, Sichuan University, Chengdu, Sichuan, China.
⁴³ State Key Laboratory of Hearing and Balance Science, Beijing Institute of Technology, Beijing, China; Senior Department of Otolaryngology Head and Neck Surgery, Chinese PLA General Hospital, Chinese PLA Medical School, Beijing, China; National Clinical Research Center for Otolaryngologic Diseases, Beijing, China; Key Laboratory of Hearing Science, Ministry of Education, Beijing, China; Beijing Key Laboratory of Hearing Impairment Prevention and Treatment, Beijing, China.
⁴⁴ Department of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
⁴⁵ Virginia Merrill Bloedel Hearing Research Center, Department of Otolaryngology-Head and Neck Surgery, University of Washington, Seattle, WA, USA.
⁴⁶ Department of Otolaryngology - Head and Neck Surgery, NewYork-Presbyterian Hospital/Columbia University Medical Center, New York, NY, USA. Electronic address: lrl2125@cumc.columbia.edu.
⁴⁷ Department of Neurology, Aerospace Center Hospital, School of Life Science, Beijing Institute of Technology, Beijing, China; State Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, Jiangsu, China; Co-Innovation Center of Neuroregeneration, Nantong University, Nantong, Jiangsu, China; Department of Otolaryngology Head and Neck Surgery, Sichuan Provincial People's Hospital, University of Electronic Science and Technology of China, Chengdu, Sichuan, China; Southeast University Shenzhen Research Institute, Shenzhen, Guangdong, China. Electronic address: renjiec@seu.edu.cn.
⁴⁸ Eaton-Peabody Laboratories, Department of Otolaryngology-Head and Neck Surgery, Mass Eye & Ear/Harvard Medical School, Boston, MA, USA. Electronic address: zheng-yi_chen@meei.harvard.edu.
⁴⁹ ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China; State Key Laboratory of Brain Function and Disorders and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China; Institutes of Biomedical Sciences, Fudan University, Shanghai, China. Electronic address: yilai_shu@fudan.edu.cn.

PMID: 41135524
DOI: 10.1016/j.medj.2025.100886

Abstract

Background: Hereditary hearing loss is one of the most common disabling disorders in children and lacks effective pharmacological treatments. Recent breakthroughs in OTOF gene therapy clinical trials necessitate standardized frameworks to guide emerging therapies. This study aims to establish the first international consensus on the clinical application of gene therapy for hereditary hearing loss.

Methods: A modified Delphi process was conducted from March 2024 to March 2025, involving 46 multidisciplinary experts from several countries across otology, genetics, audiology, gene therapy, and hearing rehabilitation. After a systematic literature review, as well as integration of research and clinical expertise and experience, three iterative voting rounds (two anonymous surveys and one online consensus meeting) were performed. Statements required ≥75% agreement for inclusion.

Findings: From 9,093 publications, 69 were used to draft and support the consensus statements. A total of 30 statements relevant to six domains achieved consensus on gene therapy for hereditary hearing loss, including ethical review (1 statement), patient selection criteria (12 statements), diagnosis and preoperative evaluation (9 statements), gene therapy drug delivery (4 statements), follow-up (3 statements), and post-treatment auditory and speech rehabilitation (1 statement).

Conclusions: This consensus provides the first globally endorsed framework for gene therapy in hereditary hearing loss. It standardizes clinical trial design and patient management, accelerating translation from research to practice while ensuring safety. The guidelines are immediately applicable to OTOF-related hearing loss and adaptable to other genetic forms.

Funding: This work was supported by the National Natural Science Foundation of China, the German Research Foundation (DFG) via the Cluster of Excellence, and others.

Keywords: Translation to population health; adeno-associated virus; clinical trial; expert consensus; gene therapy; hereditary hearing loss; modified Delphi method.

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Conflict of interest statement

Declaration of interests L.D.L. has received research funding from Decibel Therapeutics/Regeneron Pharmaceuticals and Amgen and has worked as an independent consultant for Akouos/Eli Lilly and the Gerson Lehrman Group. T.M. is a cofounder of OptoGenTech. R.N. has received research funding from Regeneron Pharmaceuticals/Sensorion. J.H.G. was a member of the board of Ohio Valley Voices and the scientific advisory board of Otonomy. M.B. has received research funding from Regeneron Pharmaceuticals. R.C. is the unpaid chief scientist of Otovia Therapeutics, Inc. Z.-Y.C. is a cofounder of Salubritas Therapeutics.

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International expert consensus on gene therapy for hereditary hearing loss: Based on clinical trials

Affiliations

International expert consensus on gene therapy for hereditary hearing loss: Based on clinical trials

Authors

Affiliations

Abstract

Conflict of interest statement

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