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CRISPR technologies for the treatment of Duchenne muscular dystrophy
The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated genome editing technologies have progressed remarkably in recent years, opening up the potential of precise genome editing as a therapeutic approach to treat various diseases. The CRISPR-CRISPR-associated (Cas)...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2021
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8571109/ https://www.ncbi.nlm.nih.gov/pubmed/33823301 http://dx.doi.org/10.1016/j.ymthe.2021.04.002 |
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author | Choi, Eunyoung Koo, Taeyoung |
author_facet | Choi, Eunyoung Koo, Taeyoung |
author_sort | Choi, Eunyoung |
collection | PubMed |
description | The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated genome editing technologies have progressed remarkably in recent years, opening up the potential of precise genome editing as a therapeutic approach to treat various diseases. The CRISPR-CRISPR-associated (Cas) system is an attractive platform for the treatment of Duchenne muscular dystrophy (DMD), which is a neuromuscular disease caused by mutations in the DMD gene. CRISPR-Cas can be used to permanently repair the mutated DMD gene, leading to the expression of the encoded protein, dystrophin, in systems ranging from cells derived from DMD patients to animal models of DMD. However, the development of more efficient therapeutic approaches and delivery methods remains a great challenge for DMD. Here, we review various therapeutic strategies that use CRISPR-Cas to correct or bypass DMD mutations and discuss their therapeutic potential, as well as obstacles that lie ahead. |
format | Online Article Text |
id | pubmed-8571109 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | American Society of Gene & Cell Therapy |
record_format | MEDLINE/PubMed |
spelling | pubmed-85711092022-11-03 CRISPR technologies for the treatment of Duchenne muscular dystrophy Choi, Eunyoung Koo, Taeyoung Mol Ther Review The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated genome editing technologies have progressed remarkably in recent years, opening up the potential of precise genome editing as a therapeutic approach to treat various diseases. The CRISPR-CRISPR-associated (Cas) system is an attractive platform for the treatment of Duchenne muscular dystrophy (DMD), which is a neuromuscular disease caused by mutations in the DMD gene. CRISPR-Cas can be used to permanently repair the mutated DMD gene, leading to the expression of the encoded protein, dystrophin, in systems ranging from cells derived from DMD patients to animal models of DMD. However, the development of more efficient therapeutic approaches and delivery methods remains a great challenge for DMD. Here, we review various therapeutic strategies that use CRISPR-Cas to correct or bypass DMD mutations and discuss their therapeutic potential, as well as obstacles that lie ahead. American Society of Gene & Cell Therapy 2021-11-03 2021-04-03 /pmc/articles/PMC8571109/ /pubmed/33823301 http://dx.doi.org/10.1016/j.ymthe.2021.04.002 Text en © 2021 The Authors https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). |
spellingShingle | Review Choi, Eunyoung Koo, Taeyoung CRISPR technologies for the treatment of Duchenne muscular dystrophy |
title | CRISPR technologies for the treatment of Duchenne muscular dystrophy |
title_full | CRISPR technologies for the treatment of Duchenne muscular dystrophy |
title_fullStr | CRISPR technologies for the treatment of Duchenne muscular dystrophy |
title_full_unstemmed | CRISPR technologies for the treatment of Duchenne muscular dystrophy |
title_short | CRISPR technologies for the treatment of Duchenne muscular dystrophy |
title_sort | crispr technologies for the treatment of duchenne muscular dystrophy |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8571109/ https://www.ncbi.nlm.nih.gov/pubmed/33823301 http://dx.doi.org/10.1016/j.ymthe.2021.04.002 |
work_keys_str_mv | AT choieunyoung crisprtechnologiesforthetreatmentofduchennemusculardystrophy AT kootaeyoung crisprtechnologiesforthetreatmentofduchennemusculardystrophy |