Human gene therapyApr 29, 2023

Improving Gene Editing with CRISPR/Cas9 to Treat Muscular Dystrophy

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 16, 2026

Abstract

Recent advances in CRISPR/Cas9 genome editing show potential for treating muscular dystrophies (MDs).

CRISPR/Cas9 techniques have demonstrated varying success in animal models of MD, including Duchenne MD and myotonic dystrophy type 1.
Innovative approaches aim to enhance precision genetic editing for better therapeutic outcomes.
Challenges remain in targeting satellite cells and improving editing efficiency in skeletal and cardiac muscle tissue.
Delivery vehicle enhancements and managing the host immunogenic response are critical for advancing these therapies.
Further research is necessary to progress CRISPR/Cas9 applications in MD toward clinical trials.

Simplified

Muscular dystrophies (MDs) comprise a diverse group of inherited disorders characterized by progressive muscle loss and weakness. Given the genetic etiology underlying MDs, researchers have explored the potential of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) genome editing as a therapeutic intervention, resulting in significant advances. Here, we review recent progress on the use of CRISPR/Cas9 genome editing as a potential therapy for MDs. Significant strides have been made in this realm, made possible through innovative techniques such as precision genetic editing by modified forms of CRISPR/Cas9. These approaches have shown varying degrees of success in animal models of MD, including Duchenne MD, congenital muscular dystrophy type 1A, and myotonic dystrophy type 1. Even so, there are several challenges facing the development of CRISPR/Cas9-based MD therapies, including the targeting of satellite cells, improved editing efficiency in skeletal and cardiac muscle tissue, delivery vehicle enhancements, and the host immunogenic response. Although more work is needed to advance CRISPR/Cas9 genome editing past the preclinical stages, its therapeutic potential for MD is extremely promising and justifies concentrated efforts to move into clinical trials.

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Improving Gene Editing with CRISPR/Cas9 to Treat Muscular Dystrophy

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You found one interesting study. We’ll send the next 7.

What Lands in Your Inbox Each Week:

Recent issues from the crispr gene editing brief