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. 2020 Jul 9:14:183.
doi: 10.3389/fncel.2020.00183. eCollection 2020.

A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome

Lucy S French  1 , Carla B Mellough  1 , Fred K Chen  1   2   3 , Livia S Carvalho  1
Affiliations

A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome

Lucy S French et al. Front Cell Neurosci. .

Abstract

Usher syndrome is a genetic disorder causing neurosensory hearing loss and blindness from retinitis pigmentosa (RP). Adaptive techniques such as braille, digital and optical magnifiers, mobility training, cochlear implants, or other assistive listening devices are indispensable for reducing disability. However, there is currently no treatment to reduce or arrest sensory cell degeneration. There are several classes of treatments for Usher syndrome being investigated. The present article reviews the progress this research has made towards delivering commercial options for patients with Usher syndrome.

Keywords: adeno-associated virus; antisense oligonucleotides; cell therapy; gene editing; gene therapy; ipscs; usher syndrome.

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Figures

Figure 1
Figure 1
Overview of the different therapeutic approaches for Usher syndrome.
See this image and copyright information in PMC

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