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Debilitating Muscular Disease Common In Boys Could Be Curbed By Rewriting Genetic Code

UT Southwestern Medical Center
The image on the left shows heart muscle that is missing the dystrophin molecule. The image on the right shows heart muscle corrected by editing with CRISPR-Cpf1. Areas of red are dystrophin.

A new gene-editing technique developed in North Texas shows promise in stunting Duchenne muscular dystrophy, a severe genetic disorder that affects about one in every 5,000 boys.

Duchenne muscular dystrophy typically passes from a mother to her son; girls are rarely affected. And, kids with the disorder typically don't live past their 20s. [KERA followed Chance Hawkins, who has the disease, in its American Graduate series, Class of '17.] 

Eric Olson, chairman of the molecular biology department at UT Southwestern explains how CRISPR technology allows scientists to rewrite genetic code, which could correct the mutation that causes the disorder.

Interview Highlights: Eric Olson...

...On Duchenne muscular dystrophy: Duchenne muscular dystrophy often abbreviated as DMD is a disease that affects all the muscles in the body, the skeletal muscle and the heart. It cause muscles to degenerate and ultimately leads to boys being confined to wheelchairs.

This disease is caused by mutations in one gene called dystrophin — that's the largest gene in the entire body. There are thousands of different mutations in that gene that can cause this disease.

...On CRISPR and how it was used in this research: CRISPR is a revolutionary new technology that now enables scientists to manipulate or rewrite the genetic code. My laboratory had worked on muscle diseases including Duchenne muscular dystrophy for decades, but really it was difficult make any headway.

When we read about the power of CRISPR to change a DNA sequence, we thought to ourselves: "How could we apply this to DMD, the devastating disease that has defied all therapies?"

So, we began with mice that have muscular dystrophy and we asked: "Could we fix the mutation in these mice that causes the disease?" And we were stunned and exhilarated to see how efficiently we were able to fix the disease in these mice.

...On this treatment's potential for humans: It's still a quite a ways off. We are testing the technology now in mice and other animals. So far, every indication that we've had says that it is safe and effective. But, you have to keep in mind that this is a completely new technology, and it has enormous power and potential. It's incumbent upon us as scientists to be cautious and respectful and to move forward in a very systematic way. If things continue to work as well as they have to date, that within a few years we might be ready to begin to think about how we could advance this into patients.

Eric Olson and his team's research appears in the journal Science Advances. 

This interview has been edited for brevity and clarity.