Cargando…

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...

Descripción completa

Detalles Bibliográficos
Autores principales: Wu, Junhao, Tao, Yong, Deng, Di, Meng, Zhaoli, Zhao, Yu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
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
_version_ 1785044956962881536
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
work_keys_str_mv AT wujunhao theapplicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT taoyong theapplicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT dengdi theapplicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT mengzhaoli theapplicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT zhaoyu theapplicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT wujunhao applicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT taoyong applicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT dengdi applicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT mengzhaoli applicationsofcrisprcasmediatedgenomeeditingingenetichearingloss
AT zhaoyu applicationsofcrisprcasmediatedgenomeeditingingenetichearingloss