CRISPR gene editing is reshaping the future of treatment for Duchenne muscular dystrophy (DMD), a severe genetic disease that weakens muscles and shortens life expectancy, according to a review published recently in Degenerative Neurological and Neuromuscular Disease.
Scientists are increasingly optimistic about CRISPR’s ability to correct the mutations that cause DMD, potentially turning a fatal diagnosis into a manageable condition. For patients, this may lead to better muscle function, extended mobility and longer lives.
DMD results from mutations in a gene responsible for dystrophin, a protein critical for muscle health. Without it, muscles steadily deteriorate, leading to loss of movement, heart problems and difficulties with breathing. Currently available treatments — including steroids, exon skipping drugs and gene therapies — help slow symptoms but don’t cure the disease. CRISPR, however, aims to fix the problem at its root by repairing the faulty gene itself.
“Advances in the precision, safety, and delivery of CRISPR systems are likely to address current challenges, such as efficient systemic delivery, consistent editing across affected tissues, and minimizing potential risks like off-target effects and immunogenicity,” stated the review’s authors. “As the field continues to evolve, the collaboration of scientists, clinicians, and regulatory agencies will be paramount in navigating the complexities of gene-editing therapies.”
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Preclinical studies using CRISPR have shown the ability to restore expression of dystrophin in human cells and animals, including mice and dogs. One approach, called single-cut exon editing, uses the body’s natural repair system to skip over faulty exons in the gene. This could benefit up to 80% of patients with DMD. Another method, double-cut exon deletion, removes problematic gene sections entirely and may apply to 85% of cases. These strategies show how gene editing could transform a severe form of DMD into a milder version, such as Becker muscular dystrophy.
Despite the promise, challenges remain. The body’s immune response can interfere with gene editing, and editing efficiency must improve to be effective in all muscle tissues, including the heart. Scientists are also developing safer tools to reduce unwanted changes elsewhere in the DNA. Still, CRISPR’s modular design and precision make it more flexible than older gene-editing technologies.
Emerging innovations such as homology-independent targeted integration and base editing further expand what’s possible. In one recent study, full-length dystrophin was restored in mice after CRISPR corrected a missing exon. Researchers also hope to increase levels of utrophin, a related protein, to support muscle health in all patients, regardless of their specific gene mutation.
For families affected by DMD, the steady progress in CRISPR therapies brings a new level of hope. While more research and clinical testing are needed, gene editing may one day offer not just symptom relief but a genuine cure.
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