先天性耳聋基因治疗药物

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全球首个!基因治疗先天性耳聋效果首次被证实优于人工耳蜗
Di Yi Cai Jing· 2025-07-24 07:58
Core Viewpoint - Cochlear implants are currently the gold standard for treating severe to profound sensorineural hearing loss, but they have limitations in restoring natural hearing and improving speech perception in noisy environments and music [1][3] Group 1: Current Treatment Landscape - Cochlear implants are the only clinical option for congenital deafness, but advancements in gene therapy may provide new treatment methods in the future [1][3] - Hearing loss is one of the most common sensory disabilities globally, affecting approximately 20% of the population, with around 26 million congenital deafness patients [3] Group 2: Gene Therapy Research - A recent study led by Professor Shu Yilai from Fudan University compared gene therapy targeting the OTOF protein with cochlear implants in 72 children, showing that gene therapy resulted in better outcomes in multiple auditory perception dimensions [4] - The study included 11 children who received gene therapy and 61 who received cochlear implants, assessing various factors such as hearing thresholds, speech tests, music perception, and quality of life over a year [4] Group 3: Future Implications - The results indicated that children who underwent gene therapy showed stable hearing recovery and surpassed cochlear implant recipients in noise speech recognition, music pitch accuracy, and auditory processing capabilities [4] - The gene therapy for congenital deafness is expected to transition into clinical applications, potentially transforming treatment approaches for hearing loss [4]