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Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss

Kamakshi Bankoti, Charles Generotti, Tiffany P. Hwa, Lili Wang, Bert W. O’Malley, Daqing Li

2021Molecular Therapy — Methods & Clinical Development34 citationsDOIOpen Access PDF

Abstract

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.

Topics & Concepts

Sensorineural hearing lossHearing lossInner earTransgeneVestibular systemGenetic enhancementMedicineAudiologyNeuroscienceBiologyGeneGeneticsHearing, Cochlea, Tinnitus, GeneticsRNA regulation and diseaseRetinal Development and Disorders
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