New hope for Duchenne muscular dystrophy (DMD) patients? CRISPR cured a mouse — and humans could be next

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Credit: Irvine/NIST
Credit: Irvine/NIST

DMD is a fatal — and currently incurable — genetic condition that causes the body’s muscles to deteriorate over time. 

The disease is always caused by errors in the gene for a muscle protein, called dystrophin. But for 10-15% of patients… the problem is caused by a duplication mutation, where part of the gene appears twice.

[Researcher Ronald] Cohn’s team injected [a model] mouse with a CRISPR molecule, designed to eliminate the mutation and (hopefully) restore the normal function of the gene.

The results were incredible.

Before and after pictures show a dramatic difference between the mouse’s muscle strength and condition. After the CRISPR treatment, signs of weakened muscles disappeared.

Not long ago, gene therapy became a promising treatment for Duchenne muscular dystrophy. But traditional gene therapy is a different process than gene editing. A gene therapy treatment works by injecting harmless, gene-carrying virus into the body to deliver a new, functional copy of a faulty gene. For DMD, that faulty gene is the dystrophin gene.

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In the case of DMD caused by a duplication mutation, CRISPR can simply snip away the harmful duplicate gene, which is much simpler than delivering a new gene or replacing the old.

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