Authors
Fan X, Gao Z, Zhong J, Chen Y, Chen X, Landegger LD, Moser T, Zeng FG, Sun Y, Jin X, Nash R, Chien WW, Jiang D, Greinwald JH, Bance M, Rodriguez MM, Lee SY, Feng G, Yang H, Wu CC, Xu L, Yuan W, Feng Y, Zhao Y, Vona BC, Stenzke N, Beutner D, Amin N, Arwyn-Jones J, Chandrasekeharan D, Shi D, Zhang D, Yang J, Qi J, Wang Q, Yin Y, Cheng YF, Tao Y, Yu Y, Wang D, Jiang L, Guo L, Chen L, Cheng X, Cui C, Lv J, Han S, Wang W, Li Y, Gao X, Liu XZ, Zha D, Shi H, Chen B, Wang Q, Yuan H, Yang S, Yin S, Wu H, Wang Z, Li H, Rubinstein JT, Lustig LR, Chai R, Chen ZY, Shu Y
Journal
Med
Citation
Med 2025. accepted manuscript.
Abstract
Hereditary hearing loss, one of the most common newborns disabling disorders, lacks effective pharmacological treatments despite cochlear implants being the long-standing treatment. Recent successes in OTOF gene therapy clinical trials highlight an urgent need for standardized clinical frameworks. Here, utilizing a modified Delphi method, we established the first international consensus on gene therapy for hereditary hearing loss among 46 multidisciplinary experts in otology, audiology, genetics, gene therapy, and hearing rehabilitation through a systematic literature review of 65 key publications, integration of research and clinical expertise and experience, and three rounds of voting from March 2024 to March 2025. Thirty statements achieved ≥ 75% agreement across six domains, 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). This consensus provides critical guidance for optimizing and standardizing clinical implementation of gene therapy for hereditary hearing loss not only for OTOF-related hearing loss but also for other genetic forms, accelerating the translation of inner ear gene therapies into clinical practice while ensuring patient safety and benefit.
DOI
No doi available.
