CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies
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(Published version)
Date
2023
Authors
Chey, Y.C.J.
Arudkumar, J.
Aartsma‐Rus, A.
Adikusuma, F.
Thomas, P.Q.
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Journal article
Citation
WIREs: Mechanisms of Disease, 2023; 15(1):e1580-1-e1580-26
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Yu C. J. Chey, Jayshen Arudkumar, Annemieke Aartsma-Rus, Fatwa Adikusuma, Paul Q. Thomas
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Abstract
CRISPR gene-editing technology creates precise and permanent modifications to DNA. It has significantly advanced our ability to generate animal disease models for use in biomedical research and also has potential to revolutionize the treatment of genetic disorders. Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disease that could potentially benefit from the development of CRISPR therapy. It is commonly associated with mutations that disrupt the reading frame of the DMD gene that encodes dystrophin, an essential scaffolding protein that stabilizes striated muscles and protects them from contractile-induced damage. CRISPR enables the rapid generation of various animal models harboring mutations that closely simulates the wide variety of mutations observed in DMD patients. These models provide a platform for the testing of sequence-specific interventions like CRISPR therapy that aim to reframe or skip DMD mutations to restore functional dystrophin expression.
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First published: 31 July 2022
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© 2022 The Authors. WIREs Mechanisms of Disease published by Wiley Periodicals LLC. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made.