Human gene therapyApr 15, 2023

Gene Editing Treatment for Duchenne Muscular Dystrophy

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Mar 16, 2026

Abstract

Genome-editing technologies have successfully corrected DMD-causing mutations in human cells, mice, and large animal models.

Duchenne muscular dystrophy (DMD) results from mutations in the dystrophin gene, leading to muscle degeneration.
Current treatments do not provide a cure for DMD despite the known genetic causes.
CRISPR-Cas9 gene editing has shown potential in correcting these mutations.
A one-time CRISPR-based treatment could restore normal gene function in affected muscles.
Challenges include improving gene editing precision, ensuring effective delivery to all muscles, and managing immune responses.

Simplified

Duchenne muscular dystrophy (DMD) is a debilitating genetic disorder that results in progressive muscle degeneration and premature death. DMD is caused by mutations in the gene encoding dystrophin protein, a membrane-associated protein required for maintenance of muscle structure and function. Although the genetic mutations causing the disease are well known, no curative therapies have been developed to date. The advent of genome-editing technologies provides new opportunities to correct the underlying mutations responsible for DMD. These mutations have been successfully corrected in human cells, mice, and large animal models through different strategies based on CRISPR-Cas9 gene editing. Ideally, CRISPR-editing could offer a one-time treatment for DMD by correcting the genetic mutations and enabling normal expression of the repaired gene. However, numerous challenges remain to be addressed, including optimization of gene editing, delivery of gene-editing components to all the muscles of the body, and the suppression of possible immune responses to the CRISPR-editing therapy. This review provides an overview of the recent advances toward CRISPR-editing therapy for DMD and discusses the opportunities and the remaining challenges in the path to clinical translation.

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Funding

Competing interests

E.N.O. is a consultant for Vertex Pharmaceuticals and Tenaya Therapeutics. The other authors declare no competing interests.

PubMed

Gene Editing Treatment for Duchenne Muscular Dystrophy

Abstract

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Abstract

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Funding

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Related papers

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