复旦大学基因治疗耳聋临床研究登上Cell子刊封面

Core Viewpoint - The research highlights the effectiveness and safety of AAV-mediated gene therapy for patients with DFNB9, a type of autosomal recessive deafness, demonstrating significant improvements in hearing recovery and language perception abilities [3][4][9]. Group 1: Clinical Research Findings - The study published in The Lancet and Nature Medicine confirms the positive outcomes of gene therapy for DFNB9 patients, showing robust hearing recovery and improvements in auditory processing [3][4]. - A longitudinal analysis of auditory characteristics post-gene therapy revealed significant changes in auditory brainstem response (ABR) and auditory steady-state response (ASSR), indicating reliable objective tools for assessing hearing recovery [7][13]. Group 2: Key Metrics and Results - All participants exhibited clear ABR V waves within 13 weeks post-treatment, with some showing ABR I and III waves by week 52, indicating positive changes in the auditory pathway [11]. - The latency of ABR V wave at 85 dB stimulus intensity decreased significantly from 9.220 milliseconds at 4 weeks to 8.190 milliseconds at 52 weeks, with an upward trend in V wave amplitude [11]. - A significant correlation was found between the thresholds of PTA, ABR, and ASSR within the 0.5-4 kHz frequency range, enhancing the understanding of the relationship between these auditory assessments [11][13]. Group 3: Implications for Future Research - The study emphasizes the importance of understanding the physiological mechanisms behind hearing recovery, including synaptic vesicle release in inner ear hair cells and central auditory pathway neuroplasticity [3][9]. - Ongoing clinical trials (ChiCTR2200063181) aim to further explore the auditory characteristics post-gene therapy, which is crucial for optimizing monitoring and evaluation strategies for hearing recovery in DFNB9 patients [9][10].