How Gene Therapy Helped Conner Run

Short Wave

NPR

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4.7 • 6K Ratings

🗓️ 5 August 2020

⏱️ 12 minutes

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Summary

Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, it could change the lives of thousands of children with Duchenne muscular dystrophy. NPR's Jon Hamilton tells us about Conner and his family...and one of the scientists who helped develop the treatment, a pioneer in the field of gene therapy.

Transcript

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0:00.0	You're listening to Shortwave from MPR.
0:06.5	Connor Curran was four years old when he was diagnosed with Duchenne muscular dystrophy,
0:12.1	a genetic disorder that was causing his muscles to waste away.
0:16.3	By the time Connor was in the first grade, his parents, Jessica and Christopher Curran,
0:21.5	could see that their son was struggling.
0:23.9	He pulled himself up the stairs.
0:25.7	He would make it past four stairs and he couldn't do the rest.
0:28.7	He could not last a full day in school.
0:31.3	The teacher would say, we let him take a little nap in the classroom and I'm thinking,
0:34.9	what?
0:35.9	Jessica remembers the advice one doctor offered the family.
0:39.1	Take your son home, love him, take him on trips while he's walking, give him a good
0:45.6	life and enjoy him because there's really not many options right now.
0:51.1	But they weren't ready to give up hope.
0:53.6	About a year after Connor's diagnosis, the Curran's heard that scientists were working
0:57.9	on a treatment, an experimental gene therapy that might be able to help.
1:03.4	The concept is very simple.
1:05.3	You're missing a gene so you're putting it back.
1:10.6	Today in the show, NPR Science correspondent John Hamilton tells us about the decades-long
1:15.4	journey to treat muscular dystrophy, the tenacious scientist, Jude Somalski, who pushed to make
1:21.9	gene therapy a reality.
1:24.3	And how his work has helped Connor Curran not just walk again, but run.
	...

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