CRISPR gene editing can be used for all manner of applications, from creating more efficient crops or heat-resistant cattle to coding GIFS into DNA. Now, scientists from the University of Texas Southwestern have showcased another potentially transformative use case for the technology: Using CRISPR to halt the progress of Duchenne muscular dystrophy (DMD) in dogs. One day, it could potentially do the same in humans, too.
DMD is the most common fatal genetic disease in children. The result of a mutation that limits the production of muscle-function protein dystrophin, it affects roughly 1 in 3,500 male births worldwide. Children who suffer with DMD often die as a result of their hearts lacking enough strength to pump, or their diaphragm being too weak to breathe. Patients also wind up in wheelchairs as the result of muscular degeneration. No effective treatment currently exists for human patients.
In their demonstration, the UT Southwestern scientists were able to use a single-cut gene-editing technique to restore the dystrophin in muscle and heart tissue by a massive 92 percent. According to experts, a 15 percent threshold could be enough to significantly help patients.
Previously, the researchers showed that it was possible to correct DMD mutations in both mice and human cells. However, in this latest breakthrough, they demonstrated that it is possible to employ this strategy in dogs, by far the largest mammals this has been applied to. Four dogs were involved in the study, all of which shared the same genetic mutation seen in DMD patients. The CRISPR gene-editing components were delivered through a harmless virus, after which it proceeded to carry out the successful gene-editing work.
More work will need to be done before this technique can be carried over to human patients in the form of a clinical trial, but that is the eventual goal. To reach this objective, the UT Southwestern team behind the research — run by regenerative science expert Dr. Eric Olson — has licensed the technology to a biotechnology company called Exonics Therapeutics. Hopefully, it won’t be too long before this work is saving lives around the world.
A paper describing the research was recently published in the journal Science.