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Review
. 2021 Apr 10;22(8):3910.
doi: 10.3390/ijms22083910.

Usher Syndrome in the Inner Ear: Etiologies and Advances in Gene Therapy

Evan M de Joya  1   2 , Brett M Colbert  1   2   3 , Pei-Ciao Tang  1 , Byron L Lam  4 , Jun Yang  5 , Susan H Blanton  1   2 , Derek M Dykxhoorn  2 , Xuezhong Liu  1   2   6
Affiliations
Review

Usher Syndrome in the Inner Ear: Etiologies and Advances in Gene Therapy

Evan M de Joya et al. Int J Mol Sci. .

Abstract

Hearing loss is the most common sensory disorder with ~466 million people worldwide affected, representing about 5% of the population. A substantial portion of hearing loss is genetic. Hearing loss can either be non-syndromic, if hearing loss is the only clinical manifestation, or syndromic, if the hearing loss is accompanied by a collage of other clinical manifestations. Usher syndrome is a syndromic form of genetic hearing loss that is accompanied by impaired vision associated with retinitis pigmentosa and, in many cases, vestibular dysfunction. It is the most common cause of deaf-blindness. Currently cochlear implantation or hearing aids are the only treatments for Usher-related hearing loss. However, gene therapy has shown promise in treating Usher-related retinitis pigmentosa. Here we review how the etiologies of Usher-related hearing loss make it a good candidate for gene therapy and discuss how various forms of gene therapy could be applied to Usher-related hearing loss.

Keywords: CRISPR; antisense oligonucleotides; retinitis pigmentosa; syndromic hearing loss.

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Conflict of interest statement

The authors declare no conflict of interest.

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