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Gene Therapy to the Retina and the Cochlea
Vision and hearing disorders comprise the most common sensory disorders found in people. Many forms of vision and hearing loss are inherited and current treatments only provide patients with temporary or partial relief. As a result, developing genetic therapies for any of the several hundred known c...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Frontiers Media S.A.
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8010260/ https://www.ncbi.nlm.nih.gov/pubmed/33815052 http://dx.doi.org/10.3389/fnins.2021.652215 |
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author | Crane, Ryan Conley, Shannon M. Al-Ubaidi, Muayyad R. Naash, Muna I. |
author_facet | Crane, Ryan Conley, Shannon M. Al-Ubaidi, Muayyad R. Naash, Muna I. |
author_sort | Crane, Ryan |
collection | PubMed |
description | Vision and hearing disorders comprise the most common sensory disorders found in people. Many forms of vision and hearing loss are inherited and current treatments only provide patients with temporary or partial relief. As a result, developing genetic therapies for any of the several hundred known causative genes underlying inherited retinal and cochlear disorders has been of great interest. Recent exciting advances in gene therapy have shown promise for the clinical treatment of inherited retinal diseases, and while clinical gene therapies for cochlear disease are not yet available, research in the last several years has resulted in significant advancement in preclinical development for gene delivery to the cochlea. Furthermore, the development of somatic targeted genome editing using CRISPR/Cas9 has brought new possibilities for the treatment of dominant or gain-of-function disease. Here we discuss the current state of gene therapy for inherited diseases of the retina and cochlea with an eye toward areas that still need additional development. |
format | Online Article Text |
id | pubmed-8010260 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-80102602021-04-01 Gene Therapy to the Retina and the Cochlea Crane, Ryan Conley, Shannon M. Al-Ubaidi, Muayyad R. Naash, Muna I. Front Neurosci Neuroscience Vision and hearing disorders comprise the most common sensory disorders found in people. Many forms of vision and hearing loss are inherited and current treatments only provide patients with temporary or partial relief. As a result, developing genetic therapies for any of the several hundred known causative genes underlying inherited retinal and cochlear disorders has been of great interest. Recent exciting advances in gene therapy have shown promise for the clinical treatment of inherited retinal diseases, and while clinical gene therapies for cochlear disease are not yet available, research in the last several years has resulted in significant advancement in preclinical development for gene delivery to the cochlea. Furthermore, the development of somatic targeted genome editing using CRISPR/Cas9 has brought new possibilities for the treatment of dominant or gain-of-function disease. Here we discuss the current state of gene therapy for inherited diseases of the retina and cochlea with an eye toward areas that still need additional development. Frontiers Media S.A. 2021-03-17 /pmc/articles/PMC8010260/ /pubmed/33815052 http://dx.doi.org/10.3389/fnins.2021.652215 Text en Copyright © 2021 Crane, Conley, Al-Ubaidi and Naash. 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 | Neuroscience Crane, Ryan Conley, Shannon M. Al-Ubaidi, Muayyad R. Naash, Muna I. Gene Therapy to the Retina and the Cochlea |
title | Gene Therapy to the Retina and the Cochlea |
title_full | Gene Therapy to the Retina and the Cochlea |
title_fullStr | Gene Therapy to the Retina and the Cochlea |
title_full_unstemmed | Gene Therapy to the Retina and the Cochlea |
title_short | Gene Therapy to the Retina and the Cochlea |
title_sort | gene therapy to the retina and the cochlea |
topic | Neuroscience |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8010260/ https://www.ncbi.nlm.nih.gov/pubmed/33815052 http://dx.doi.org/10.3389/fnins.2021.652215 |
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