Science 3 min read

Using CRISPR Gene-Editing to Correct Muscular Dystrophy

Pan Andrii /

Pan Andrii /

A team of German researchers has used the CRISPR geneediting tool to correct muscular dystrophy in pigs.

Duchenne muscular dystrophy (DMD) is a rare muscle disorder.

However, it’s one of the most common genetic condition, affecting about 1 in 3,500 male births across the world. Along with reducing the patient’s quality of life, the condition also reduced their life expectancy.

Typically, the muscles need a protein called dystrophin to regenerate. But in people with DMD, a genetic mutation removes the gene that produces dystrophin.

As a result, affected children would start showing signs of muscle weakness by age five. While they lose the ability to walk at about age 12, their heart muscles often give out before their 30s.

Since it’s a genetic condition, DMD has always been a primary target for the gene-editing tool, CRISPR. Now, researchers in Germany have successfully used the tool to correct the condition in pigs.

The team published their findings in the journal Nature Medicine.

Correcting Muscular Dystrophy in Pigs

In their experiment with pigs, the researchers used CRISPR to repair the faulty dystrophin gene. So, the animals started producing dystrophin proteins.

Although the proteins were shorter than usual, the researchers noted that they were stable and functional. As a result, the pigs became less likely to develop an irregular heartbeat, which in turn improved their life expectancy.

Besides, the treatment avoids one of the primary issues with CRISPR gene-editing, and that’s transience.

At some point, most body cells die off and become replaced by another of the same type. That means the edited cells should eventually disappear, rendering the treatment ineffective.

Fortunately, heart muscles live longer. According to the team, one-half of all myocardial cells remain functional from birth throughout the entire lifecycle of a human being.

So, performing the edit on these cells ensures that effects endure for a more extended period.

Lead author of the study, Christian Kupatt points out:

“The genome of a cell is used for protein biosynthesis as long as the cell is alive. And once a cell has been affected by the therapy, it remains correct. So if we change the genome of a myocardial cell, the correction is a long-term success, in contrast to the results of previous methods.”

This is not the first experiment to use CRISPR to correct DMD in animals.

A 2014 study used the gene-editing tool on mice with the muscular condition. Similarly, in 2017, a team of European researchers used gene therapy to treat dogs with DMD.

However, this is the first time researchers are using pigs to test the treatment. Since these animals are biologically similar to humans, clinical trials for muscular dystrophy gene treatment may be in the nearest future.

Read More: Harnessing Class 1 CRISPR Systems to Improve Human Gene Editing

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Sumbo Bello is a creative writer who enjoys creating data-driven content for news sites. In his spare time, he plays basketball and listens to Coldplay.

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