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

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

Funding

None.

Conflict of Interest

None declared.

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Global Medical Genetics