Science https://doi.org/10.1126/science.aau1549 (2018).
The deltaE50-MD dog model of Duchenne muscular dystrophy (DMD) can be treated with systemic delivery of CRISPR gene editing components that restore the expression of the dystrophin gene.
DMD is characterized by progressive muscle degeneration and atrophy and is caused by mutations in the dystrophin gene that result in its decreased expression. It has been shown that the gene can be targeted with CRISPR–Cas9 to restore its expression in mice and muscle stem cells.
The deltaE50-MD dog model of DMD has a mutation in the dystrophin gene that corresponds with a mutational hotspot in humans. Researchers were able to restore its expression in this model using CRISPR–Cas9, showing the potential for translation in humans.
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Stower, H. Gene editing for Duchenne muscular dystrophy. Nat Med 24, 1491 (2018). https://doi.org/10.1038/s41591-018-0225-1
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DOI: https://doi.org/10.1038/s41591-018-0225-1
