基因疗法后的听觉特征在患有自体逆行性聋症的患者中9
Longlong Zhang1, Dingding Dong1, Yanbo Yin1
1ENT Institute and Otorhinolaryngology Department of Eye & ENT Hospital, State Key Laboratory of Medical Neurobiology and MOE Frontiers Center for Brain Science, Fudan University, Shanghai 200031, China; Institutes of Biomedical Sciences, Fudan University, Shanghai 200032, China; NHC Key Laboratory of Hearing Medicine, Fudan University, Shanghai 200031, China.
Med (New York, N.Y.)
|May 23, 2025
概括
基因疗法对9型自体衰退性聋症 (DFNB9) 患者显示出前景. 听觉脑干反应 (ABR) 和听觉稳定状态反应 (ASSR) 有效地追踪治疗后听力恢复.
科学领域:
- 遗传学和听力学
背景情况:
- 基因疗法为自体逆行性耳聋9 (DFNB9) 提供了潜在的疗法.
- 优化患者监测需要了解基因治疗后的听力学变化.
研究的目的:
- 为了评估基因治疗后DFNB9患者的听力学变化.
- 评估听觉脑干响应 (ABR) 和听觉稳定状态响应 (ASSR) 在监测听力恢复中的有用性.
主要方法:
- 收集了基因治疗后的10名DFNB9患者的听觉数据.
- 利用了听觉脑干响应 (ABR),听觉平稳状态响应 (ASSR),扭曲产物耳声发射 (DPOAE) 和纯色调听力测量 (PTA).
主要成果:
- 在治疗后52周观察到的1kHzABR波V延迟显著下降.
- 随着时间的推移,ABR波I,III和V的积极变化被观察到.
- 在PTA,ABR和ASSR值之间发现了相关性;DPOAE没有显示显著的治疗后变化.
结论:
- ABR和ASSR是可靠的客观措施,用于评估基因治疗后DFNB9患者的听力恢复.
- 基因治疗对听觉通路产生积极影响,随着时间的推移,ABR的变化证明了这一点.
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