The cutting edge of CRISPR: A lifesaving treatment for DMD

UT Southwestern scientists are on the cusp of solving genetic diseases by snipping defective DNA.

Transcript

[Narrator] Since being diagnosed at nine years old, 26-year-old Ben Dupree has been redefining how someone with Duchenne muscular dystrophy can live.

When I was kind of diagnosed, I guess, one thing that I always kind of stuck to was that I was doing better than what the books said.

[Female Voice] Mack, come on, come here buddy.

[Narrator] What he read back then was that he wouldn't live past 20 and while he has been wheelchair-bound for most of his life, that hasn't slowed his speed for success.

For a lot of things, it's not it can't be done but that you kinda have to approach it from a different angle or differently than most people would.

[Narrator] Dupree has a Bachelor's degree in biochemistry from Southern Methodist University. His contributions to science are now having profound effects in the lab of molecular biologist, Dr. Eric Olson.

I wanna cure Duchenne muscular dystrophy. That's what I wanna do.

[Narrator] He's dedicated much of his lengthy career at UT Southwestern Medical Center to do just that.

[Ben] I really admire the drive around this research.

[Narrator] Olson has perfected cutting edge CRISPR gene editing technology that's allowed his team to halt the progression of DMD, most recently by successfully editing the inherited gene in dogs and hopefully soon a clinical trial for humans.

Ben has played an instrumental role in our research. Early on, he was our patient zero and he graciously provided us with a small blood sample. One of the most inspiring aspects of the work was the moment when Ben came to the lab and together we looked through the microscope at his own cells that came from his blood sample and they were beating in a dish and then we showed him that they were producing the dystrophin protein that his body cannot make.

Being able to see that gave me a lot of hope for the future kinda of seeing that this is a concrete thing that can happen. It could be done.

[Narrator] So what is gene editing and how does it work?

[Dr. Olson] If you want to try to conceptualize what gene editing is think about the letters in a book and in the book of life, of humans, there's three billion of those letters. Even one letter error can cause a devastating disease like Duchenne.

[Narrator] That's the reality for Dupree. One out of three billion letters in his DNA is off. The hope is that gene editing can correct it.

[Dr. Olson] If you could think about CRISPR gene editing almost as a spell checker. You can program it to find any misspelled word in the book of life and you can direct it to go there and to change that letter back to normal. So it's very powerful. It's effective and it's fast.

[Narrator] And while Dupree accepts the reality that the technology may not work fast enough for him, he's encouraged by the progress in knowledge, that it may one day help others.

The biggest thing that keeps me moving forward is really feeling that I've found a purpose, I guess, in helping other people through what I've gone through. Hey guys, it's Ben Dupree. You're watching my first Youtube video and I kind of found a way to represent the voice of someone living with Duchenne muscular dystrophy in a variety of different capacities. I hope that even if I'm not there to see really great leaps in treatments, that there'll be people being born now that will see those things.