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JAMA Neurology:舒易来团队发现,基因治疗对先天性耳聋患者自然听力的恢复,优于人工耳蜗
生物世界·2025-07-22 07:02

Core Viewpoint - The article discusses the advancements in gene therapy for congenital deafness, highlighting its superiority over traditional cochlear implants in restoring natural hearing and improving speech perception [2][12]. Group 1: Overview of Hearing Loss - Hearing loss is one of the most common sensory disabilities globally, affecting 20% of the population, with 5% experiencing disabling hearing loss [2]. - Approximately 2-3 out of every 1000 newborns are born with congenital hearing impairment, with 60% of cases linked to genetic factors [2]. Group 2: Traditional Treatments and Limitations - Cochlear implants have been the gold standard for severe hearing loss for nearly half a century, but they do not fully restore natural hearing and have low acceptance due to external device maintenance [2][12]. - There has been no clinical treatment available for congenital deafness until the emergence of gene therapy [2]. Group 3: Gene Therapy Advancements - A study led by Fudan University found that gene therapy significantly outperformed cochlear implants in restoring hearing function and speech perception in children with congenital deafness [3][6]. - The study included 11 children who received gene therapy and compared them with 61 children who received cochlear implants, assessing multiple dimensions of auditory perception over a year [6]. Group 4: Results of Gene Therapy - Among the 11 patients treated with gene therapy, 9 showed stable hearing recovery and improved speech abilities after one year [8]. - The gene therapy group scored significantly higher in various auditory perception tests compared to the cochlear implant group, indicating better auditory processing capabilities [9][10]. Group 5: Clinical Implications - The research provides evidence that gene therapy can lead to faster recovery and better quality of auditory perception compared to cochlear implants, marking a new era in precise auditory medicine [12][26]. - The findings suggest that even patients with prior cochlear implants can benefit from subsequent gene therapy, enhancing their auditory capabilities [10]. Group 6: Future Directions - The success of gene therapy for congenital deafness may pave the way for advancements in treating other rare genetic diseases, as the techniques developed can be applied to broader genetic conditions [24][25]. - A new laboratory focused on rare disease gene editing and cell therapy has been established to further research in this area, aiming to develop effective treatments for various genetic disorders [24][25].