Original Investigation

Gene Therapy vs Cochlear Implantation in Restoring Hearing Function and Speech Perception for Individuals With Congenital Deafness

JAMA Neurology 10.1001/jamaneurol.2025.2053

July 21, 2025 at 11:00 AM EDT

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Among patients with congenital deafness, is gene therapy (GT) more efficacious in auditory and speech perception than cochlear implantation (CI)?This cohort study including 72 individuals found that, globally, while patients undergoing GT only showed comparable auditory and speech perception to CI patients at 12 months after treatment, GT-only patients showed earlier improvements at 6 months after treatment. In bimodal patients evaluated at 12 months, GT outperformed CI in ambient sound detection, speech tests in noisy environments, and in-tune singing rates.The findings in this study comparing GT and CI on multidimensional auditory and speech perception offer functional evidence for future decision-making.

Corresponding Author: Yilai Shu, MD, PhD, ENT Institute and Otorhinolaryngology Department, Eye and ENT Hospital of Fudan University, Shanghai, 200031, China (yilai_shu@fudan.edu.cn).

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