Clinical practice guidelines for gene therapy to treat hereditary hearing loss

• DOI: 10.1002/inmd.20240008
• Publication Date: 2024-03-16T13:53:47Z
• Authors: Jieyu Qi; Fangzhi Tan; Liyan Zhang; Ling Lu; Hongyang Wang; Wenyan Li; Wenwen Liu; Xiaolong Fu; Zuhong He; Xiaoqiong Ding; Shan Sun; Qiaojun Fang; Yaodong Dong; Xuewei Zhu; Busheng Tong; Xianbao Cao; Min Guo; Xinmiao Fan; Qin Wang; Lu Ma; Tianhong Zhang; Yafeng Yu; Yongxin Li; Jiangang Fan; Yong Cui; Peina Wu; Hongzheng Zhang; Jie Tang; Weiwei Guo; Dingjun Zha; Fanglei Ye; Shuangba He; Wei Cao; Jianming Yang; Xiaoyun Qian; Yu Zhao; Jingwu Sun; Xiaowei Chen; Yu Sun; Ming Xia; Qiuju Wang; Huijun Yuan; Yong Feng; Weijia Kong; Shiming Yang; Haibo Wang; Maoli Duan; Xia Gao; Huawei Li; Lei Xu; Renjie Chai
• Keywords: 0303 health sciences; 03 medical and health sciences; Medical technology; R855-855.5; gene therapy; guideline; TP248.13-248.65; clinical practice; Biotechnology

AbstractHereditary deafness is a common neurosensory disorder, and 148 non‐syndromic deafness genes have been identified to date. Gene therapy has been used to treat a variety of genetic diseases, but no gene therapy drug for hereditary deafness has been approved for clinical use. At present, several clinical trials of gene therapy for hereditary deafness are underway. However, few normative documents have been issued to guide the standardization of gene therapy for hearing loss, and this document is the first global gene therapy guideline for hereditary hearing loss. The guidelines were jointly developed and drafted by experienced audiologists, virologists and biologists who are vigorously involved in inner ear gene therapy research in the Hearing, Speech and Communication Subsociety of Biophysical Society of China, Audiology Development Foundation Of China and Audiology Subsociety of Jiangsu Medical Association. These guidelines cover preclinical research and clinical practice of gene therapy for hereditary deafness, including indications, key points of pre‐clinical research, patient selection criteria, pre‐clinical preparation, drug efficacy, drug safety evaluation criteria, ethical review, etc. We hope that the guidelines will promote the standardization of clinical practice related to gene therapy for hereditary deafness in China and around the world.