How Can CRISPR Help People with Duchenne Muscular Dystrophy?

CRISPR has the power to change the lives of people living with DMD. Image courtesy of Wikipedia Commons.

CRISPR has the power to change the lives of people living with DMD. Image courtesy of Wikipedia Commons.

Layla Loew, Writer

Imagine your muscles deteriorating slowly, and painfully over time. Eventually your dying muscles would give up, and standing up becomes impossible. Imagine your child develops the same inevitable physical decline. Now, imagine there’s a way to prevent this through genetic engineering.

 

Duchenne Muscular Dystrophy is a rare genetic disorder that causes muscle loss and physical impairment that can last a few years or a lifetime. In children especially, DMD causes a gene mutation to interrupt a protein in skeletal muscle called dystrophin. This disruption causes the muscles that control heart and breathing begin to atrophy and cause death.

 

Although females can be carriers for this disorder, males are the most affected because it is X-linked. Males, therefore, have a greater chance to be affected by the DMD gene. According to a report about Duchenne Muscular Dystrophy from the National Organization For Rare Disorders states that “DMD is the most common childhood onset form of muscular dystrophy and affects males almost exclusively.”

 

The Professor of Pediatrics, Gyula Acsadi, who assisted in creating this report, estimates that about 1 in 3,500 male births appear to have DMD. Muscular dystrophy in general affects about 250,000 individuals in the United States.

 

DMD might be rare, but the impact is still devastating. However, the gene editing tool CRISPR could be used to edit out the gene in the stem cells that causes early muscle death. Tina Heman Saey, in her article for Science News for Students, “Explainer: How CRISPR Works,” she explains that CRISPR is a cheap and easy to use tool that can “quickly and efficiently tweak almost any gene in any plant or animal.” The tool has been useful for scientists to “cut DNA with CRISPR/Cas 9 to make gene changes, or mutations.”

 

In the article, “Researchers Use CRISPR To Correct Mutation in Duchenne Muscular Dystrophy Model,” from the website Genomics Research from Technology Networks, researchers from several universities conducted experiments to edit out gene mutations. The goal was to edit the mutation causing DMD and make a lifelong correction. However, there was a concern that the edited gene would eventually relapse over time, meaning the change wasn’t permanent.

 

To prevent this from happening, researchers from the University of Missouri School of Medicine, the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine, and Duke University targeted the mutation in the muscle stem cells. As a result, the cells that regenerated from the edited stem cells will have no sign of the muscular disorder. In other words, CRISPR can avoid a relapse of DMD by only regenerating edited genes.

 

A seemingly simple process like this one can improve the lives of thousands. Although this research is still in the experimental phase, the results are promising.

 

Genetic disorders are thought of to be unavoidable and incurable, but advancing genetic engineering tools could be the answer to fewer deaths and improved quality of life.