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The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss
Hearing loss (HL) can be caused by a number of different genetic factors. Non-syndromic HL refers that HL occurs as an isolated symptom in an individual, whereas syndromic HL refers that HL is associated with other symptoms or abnormalities. To date, more than 140 genes have been identified as being...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10199548/ https://www.ncbi.nlm.nih.gov/pubmed/37210555 http://dx.doi.org/10.1186/s13578-023-01021-7 |
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author | Wu, Junhao Tao, Yong Deng, Di Meng, Zhaoli Zhao, Yu |
author_facet | Wu, Junhao Tao, Yong Deng, Di Meng, Zhaoli Zhao, Yu |
author_sort | Wu, Junhao |
collection | PubMed |
description | Hearing loss (HL) can be caused by a number of different genetic factors. Non-syndromic HL refers that HL occurs as an isolated symptom in an individual, whereas syndromic HL refers that HL is associated with other symptoms or abnormalities. To date, more than 140 genes have been identified as being associated with non-syndromic HL, and approximately 400 genetic syndromes can include HL as one of the clinical symptoms. However, no gene therapeutic approaches are currently available to restore or improve hearing. Therefore, there is an urgent necessity to elucidate the possible pathogenesis of specific mutations in HL-associated genes and to investigate the promising therapeutic strategies for genetic HL. The development of the CRISPR/Cas system has revolutionized the field of genome engineering, which has become an efficacious and cost-effective tool to foster genetic HL research. Moreover, several in vivo studies have demonstrated the therapeutic efficacy of the CRISPR/Cas-mediated treatments for specific genetic HL. In this review, we briefly introduce the progress in CRISPR/Cas technique as well as the understanding of genetic HL, and then we detail the recent achievements of CRISPR/Cas technique in disease modeling and therapeutic strategies for genetic HL. Furthermore, we discuss the challenges for the application of CRISPR/Cas technique in future clinical treatments. |
format | Online Article Text |
id | pubmed-10199548 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-101995482023-05-21 The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss Wu, Junhao Tao, Yong Deng, Di Meng, Zhaoli Zhao, Yu Cell Biosci Review Hearing loss (HL) can be caused by a number of different genetic factors. Non-syndromic HL refers that HL occurs as an isolated symptom in an individual, whereas syndromic HL refers that HL is associated with other symptoms or abnormalities. To date, more than 140 genes have been identified as being associated with non-syndromic HL, and approximately 400 genetic syndromes can include HL as one of the clinical symptoms. However, no gene therapeutic approaches are currently available to restore or improve hearing. Therefore, there is an urgent necessity to elucidate the possible pathogenesis of specific mutations in HL-associated genes and to investigate the promising therapeutic strategies for genetic HL. The development of the CRISPR/Cas system has revolutionized the field of genome engineering, which has become an efficacious and cost-effective tool to foster genetic HL research. Moreover, several in vivo studies have demonstrated the therapeutic efficacy of the CRISPR/Cas-mediated treatments for specific genetic HL. In this review, we briefly introduce the progress in CRISPR/Cas technique as well as the understanding of genetic HL, and then we detail the recent achievements of CRISPR/Cas technique in disease modeling and therapeutic strategies for genetic HL. Furthermore, we discuss the challenges for the application of CRISPR/Cas technique in future clinical treatments. BioMed Central 2023-05-20 /pmc/articles/PMC10199548/ /pubmed/37210555 http://dx.doi.org/10.1186/s13578-023-01021-7 Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data. |
spellingShingle | Review Wu, Junhao Tao, Yong Deng, Di Meng, Zhaoli Zhao, Yu The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss |
title | The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss |
title_full | The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss |
title_fullStr | The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss |
title_full_unstemmed | The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss |
title_short | The applications of CRISPR/Cas-mediated genome editing in genetic hearing loss |
title_sort | applications of crispr/cas-mediated genome editing in genetic hearing loss |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10199548/ https://www.ncbi.nlm.nih.gov/pubmed/37210555 http://dx.doi.org/10.1186/s13578-023-01021-7 |
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