Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeuticsFeb 15, 2022

Gene Therapy Using CRISPR-Cas9 to Treat Duchenne Muscular Dystrophy

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 16, 2026

Abstract

CRISPR-Cas9 technology has potential applications for correcting mutations in the Duchenne muscular dystrophy gene, which affect 100% of patients.

The majority of Duchenne muscular dystrophy cases involve exon deletions (70%) and point mutations (30%).
CRISPR-Cas9 technology is evolving to become more precise with the introduction of base editing and prime editing.
Current research focuses on addressing off-target effects, ensuring efficient delivery to muscle tissues, and managing immune responses to the technology.
Recent strategies aim to restore dystrophin expression in laboratory settings and animal models of Duchenne muscular dystrophy.

Simplified

Discovery of the CRISPR-Cas (clustered regularly interspaced short palindromic repeat, CRISPR-associated) system a decade ago has opened new possibilities in the field of precision medicine. CRISPR-Cas was initially identified in bacteria and archaea to play a protective role against foreign genetic elements during viral infections. The application of this technique for the correction of different mutations found in the Duchenne muscular dystrophy (DMD) gene led to the development of several potential therapeutic approaches for DMD patients. The mutations responsible for Duchenne muscular dystrophy mainly include exon deletions (70% of patients) and point mutations (about 30% of patients). The CRISPR-Cas 9 technology is becoming increasingly precise and is acquiring diverse functions through novel innovations such as base editing and prime editing. However, questions remain about its translation to the clinic. Current research addressing off-target editing, efficient muscle-specific delivery, immune response to nucleases, and vector challenges may eventually lead to the clinical use of the CRISPR-Cas9 technology. In this review, we present recent CRISPR-Cas9 strategies to restore dystrophin expression in vitro and in animal models of DMD.

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Gene Therapy Using CRISPR-Cas9 to Treat Duchenne Muscular Dystrophy

Abstract

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