Genetic Medicine for Hearing Loss: OTOF as Exemplar

Abstract

Millions of people worldwide have disabling hearing loss because one of their genes generates an incorrect version of some specific protein the ear requires for hearing. In many of these cases, delivering the correct version of the gene to a specific target cell within the inner ear has the potential to restore cochlear function to enable high-acuity physiologic hearing. Purpose: In this review, we outline our strategy for the development of genetic medicines with the potential to treat hearing loss. We will use the example of otoferlin gene (OTOF)-mediated hearing loss, a sensorineural hearing loss due to autosomal recessive mutations of the OTOF gene.

Keywords

Ear, Inner - Genetic Testing - Genetic Therapy - Hearing Loss

Disclaimer

Any mention of a product, service, or procedure in the Journal of the American Academy of Audiology does not constitute an endorsement of the product, service, or procedure by the American Academy of Audiology.

^* Co-first authors.

Publication History

Received: 09 November 2020

Accepted: 19 January 2021

Article published online:
24 May 2022

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