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CC BY 4.0 · Glob Med Genet 2024; 11(04): 349-357
DOI: 10.1055/s-0044-1791803
Review Article

The Role of CRISPR/Cas9 in Revolutionizing Duchenne's Muscular Dystrophy Treatment: Opportunities and Obstacles

Ahsan Ali
1   Faculty of Health, Plymouth University Peninsula Medical School, Plymouth, United Kingdom
,
Md Yakeen Rahman
2   University of Plymouth Faculty of Heath, Plymouth, United Kingdom
,
Danish Sheikh
2   University of Plymouth Faculty of Heath, Plymouth, United Kingdom
› Author Affiliations Funding None.
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Abstract

Duchenne's muscular dystrophy (DMD) is a severe X-linked disorder characterized by progressive muscle degeneration, leading to loss of ambulation, respiratory failure, and premature death. It affects approximately 1 in 3,500 live male births and is caused by mutations in the dystrophin gene, which impairs muscle fiber stability. Current treatments are limited to managing symptoms and slowing disease progression, with no curative therapies available. The advent of CRISPR/Cas9 gene-editing technology has introduced a promising approach for directly correcting the genetic mutations responsible for DMD. This review explores the potential of CRISPR/Cas9 as a transformative therapy for DMD, highlighting its successes in preclinical models, the challenges associated with its delivery, and the obstacles to its clinical application. While preclinical studies demonstrate the efficacy of CRISPR/Cas9 in restoring dystrophin expression and improving muscle function, significant hurdles remain, including optimizing delivery methods and ensuring long-term safety.

Keywords

CRISPR/Cas9 - Duchenne's muscular dystrophy - gene editing - gene therapy - preclinical models


Publication History

Article published online:
18 October 2024

© 2024. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution License, permitting unrestricted use, distribution, and reproduction so long as the original work is properly cited. (https://creativecommons.org/licenses/by/4.0/)

Georg Thieme Verlag KG
Stuttgart · New York

 

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