CRISPR shows promise for treating muscular dystrophy

BY JOSEPH SCALISE
Posted on FEB 13, 2019

A group of scientists have discovered yet another disease that CRISPR can effectively combat.

Researchers from the University of Texas Southwestern Medical Center have used the gene editing technology CRISPR to cure muscular dystrophy in dogs, which suggests the technology could one day be ported over to humans.

In the study, the team used CRISPR on four beagles bred with the gene that triggers the disease and found that the technology is able to correct thegenetic defect.
Muscular dystrophy comes about through mutations in the dystrophin gene, which codes for a protein that allows normal muscle function. That causes large problems in humans, including weak muscles, as well as heart and respiratory failure.

In the study, the team corrected the mutated dystrophin gene in the dogs by using CRISPR to splice out the dangerous section. Theydirectly injected CRISPR technology into two of the dogs through their muscles and two of the dogs through their bloodstream to see how it affected different parts of the body.

The injections caused the canines' muscle cells throughout their body to create healthy dystrophin protein at anywhere from 3 percent to 90 percent of the normal levels eight weeks after the procedure. That is significant because even raising those levels 15 percent of normal levels in people would completely change their lives and enable them to function.

This study is important, not just for muscular dystrophy treatment, but also because it is yet another example of CRISPR helping to fight a human disease. That furthers its potential and reveals that, despite setbacks, the technology could be a big help in the future.

While the results of the study came from a few dogs, they do show a lot of promise. The next step is to conduct studies on larger animals and see how their cells respond.

"We are going for a cure, not a treatment," explained study co-author Eric Olson, a researcher fromthe University of Texas Southwestern Medical Center, according to TIME. "All of the other therapies so far for Duchenne muscular dystrophy have treated the symptoms and consequences of the disease. This is going right at the root cause of the genetic mutation."

The new study is published in the journal Science.

 

 

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