Molecular Therapy - Methods & Clinical Development ( IF 4.6 ) Pub Date : 2021-03-10 , DOI: 10.1016/j.omtm.2021.03.005 Kamakshi Bankoti , Charles Generotti , Tiffany Hwa , Lili Wang , Bert W. O’Malley , Daqing Li
There is growing attention and effort focused on treating the root cause of sensorineural hearing loss rather than managing associated secondary characteristic features. With recent substantial advances in understanding sensorineural hearing-loss mechanisms, gene delivery has emerged as a promising strategy for the biological treatment of hearing loss associated with genetic dysfunction. There are several successful and promising proof-of-principle examples of transgene deliveries in animal models; however, there remains substantial further progress to be made in these avenues before realizing their clinical application in humans. Herein, we review different aspects of development, ongoing preclinical studies, and challenges to the clinical transition of transgene delivery of the inner ear toward the restoration of lost auditory and vestibular function.
中文翻译:
腺相关病毒内耳基因治疗感音神经性耳聋的研究进展与挑战
越来越多的注意力和精力集中在治疗感觉神经性听力损失的根本原因上,而不是管理相关的次要特征。随着最近对理解感音神经性听力损失机制的重大进展,基因传递已成为一种有前途的策略,用于对与遗传功能障碍相关的听力损失进行生物治疗。在动物模型中,有几个成功且很有希望的转基因传递原理证明实例。然而,在实现它们在人类中的临床应用之前,在这些途径上仍然有实质性的进一步进展。在此,我们回顾了开发的各个方面,正在进行的临床前研究,