OTOF耳聋基因疗法
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登上Cell头条:我国学者领衔,全球首个遗传性耳聋基因治疗国际专家共识发布
生物世界· 2025-10-26 01:00
Core Viewpoint - The article discusses the global consensus on gene therapy for hereditary hearing loss, marking a significant milestone in the field and providing a standardized framework for clinical applications [3][10][14]. Group 1: Consensus Development - The consensus was led by a team from Fudan University and involved 46 experts from various countries, taking over a year to develop [5][7]. - It was established using a modified Delphi method, resulting in 30 consensus statements covering key areas such as ethical review, patient selection, preoperative diagnosis, gene therapy delivery, postoperative follow-up, and auditory rehabilitation [7][14]. Group 2: Clinical Significance - According to WHO, over 430 million people globally suffer from disabling hearing loss, with genetic factors accounting for 60% of congenital hearing loss cases [10]. - The consensus aims to standardize clinical trials and patient management for gene therapy, particularly for OTOF-related hearing loss, and is expected to provide important references for clinicians, researchers, and regulatory bodies [15]. Group 3: Expert Opinions - Experts emphasized that this consensus represents a crucial step in establishing a unified framework for clinical trials in gene therapy for hearing loss, ensuring safety and scientific rigor [14]. - The consensus is seen as a potential game-changer in the treatment landscape for hereditary hearing loss, filling a significant gap in the guidance for gene therapy in this area [14][15].