Please use this identifier to cite or link to this item:
https://hdl.handle.net/2440/136040
Citations | ||
Scopus | Web of Science® | Altmetric |
---|---|---|
?
|
?
|
Type: | Journal article |
Title: | CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies |
Author: | Chey, Y.C.J. Arudkumar, J. Aartsma‐Rus, A. Adikusuma, F. Thomas, P.Q. |
Citation: | WIRES MECHANISMS OF DISEASE, 2023; 15(1):e1580-1-e1580-26 |
Publisher: | Wiley |
Issue Date: | 2023 |
ISSN: | 2692-9368 2692-9368 |
Statement of Responsibility: | Yu C. J. Chey, Jayshen Arudkumar, Annemieke Aartsma-Rus, Fatwa Adikusuma, Paul Q. Thomas |
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. |
Keywords: | CRISPR/Cas9; CRISPR therapy; Duchenne muscular dystrophy; animal models; mice models |
Description: | First published: 31 July 2022 |
Rights: | © 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. |
DOI: | 10.1002/wsbm.1580 |
Published version: | http://dx.doi.org/10.1002/wsbm.1580 |
Appears in Collections: | Genetics publications |
Files in This Item:
File | Description | Size | Format | |
---|---|---|---|---|
hdl_136040.pdf | Published version | 4.01 MB | Adobe PDF | View/Open |
Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.