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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 disabili...

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Detalles Bibliográficos
Autores principales: French, Lucy S., Mellough, Carla B., Chen, Fred K., Carvalho, Livia S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7363968/
https://www.ncbi.nlm.nih.gov/pubmed/32733204
http://dx.doi.org/10.3389/fncel.2020.00183
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author French, Lucy S.
Mellough, Carla B.
Chen, Fred K.
Carvalho, Livia S.
author_facet French, Lucy S.
Mellough, Carla B.
Chen, Fred K.
Carvalho, Livia S.
author_sort French, Lucy S.
collection PubMed
description 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.
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spelling pubmed-73639682020-07-29 A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome French, Lucy S. Mellough, Carla B. Chen, Fred K. Carvalho, Livia S. Front Cell Neurosci Cellular Neuroscience 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. Frontiers Media S.A. 2020-07-09 /pmc/articles/PMC7363968/ /pubmed/32733204 http://dx.doi.org/10.3389/fncel.2020.00183 Text en Copyright © 2020 French, Mellough, Chen and Carvalho. http://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Cellular Neuroscience
French, Lucy S.
Mellough, Carla B.
Chen, Fred K.
Carvalho, Livia S.
A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome
title A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome
title_full A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome
title_fullStr A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome
title_full_unstemmed A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome
title_short A Review of Gene, Drug and Cell-Based Therapies for Usher Syndrome
title_sort review of gene, drug and cell-based therapies for usher syndrome
topic Cellular Neuroscience
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7363968/
https://www.ncbi.nlm.nih.gov/pubmed/32733204
http://dx.doi.org/10.3389/fncel.2020.00183
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