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

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 disabilit...

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Published inFrontiers in cellular neuroscience Vol. 14; p. 183
Main Authors French, Lucy S., Mellough, Carla B., Chen, Fred K., Carvalho, Livia S.
Format Journal Article
LanguageEnglish
Published Lausanne Frontiers Research Foundation 09.07.2020
Frontiers Media S.A
Subjects
adeno-associated virus
Blindness
Brain-derived neurotrophic factor
cell therapy
Cellular Neuroscience
Cochlea
Congenital diseases
Deafness
Degeneration
Deoxyribonucleic acid
DNA
gene editing
gene therapy
Genes
Genetic disorders
Genetic engineering
Hearing loss
ipscs
Lipids
Mutation
Nanoparticles
Photoreceptors
Polyethylene glycol
Proteins
Respiratory distress syndrome
Retina
Retinitis
Retinitis pigmentosa
Transplants & implants
usher syndrome
Vectors (Biology)
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Summary: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 that research has made toward delivering commercial options for patients with Usher syndrome.
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Specialty section: This article was submitted to Cellular Neuropathology, a section of the journal Frontiers in Cellular Neuroscience
Reviewed by: Nicholas D. Mazarakis, Imperial College London, United Kingdom; Wei Xiong, Tsinghua University, China
Edited by: Raymond Ching-Bong Wong, Centre for Eye Research Australia, Australia
ISSN:1662-5102
1662-5102
DOI:10.3389/fncel.2020.00183