How Scientists Made The First Gene-Editing Treatment For A Baby

Science Friday

Science Friday and WNYC Studios

Life Sciences, Wnyc, Science, Earth Sciences, Natural Sciences, Friday

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🗓️ 25 June 2025

⏱️ 19 minutes

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Summary

Last month, a baby got the world’s first personalized gene-editing treatment. What will this mean for millions of others with genetic diseases?

Transcript

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0:00.0	Hey, this is Flor Lichtenen, and you're listening to Science Friday.
0:07.0	Today in the podcast, the story of a medical breakthrough many years in the making.
0:13.3	One of my biggest fears in this whole process has been giving false hope.
0:16.2	And so I wanted to be honest about all the things that could go wrong.
0:24.5	Last. wanted to be honest about all the things that could go wrong. Last month, an historic first.
0:27.2	Scientists created the first personalized gene-edited treatment for a baby, who was born
0:32.4	with a rare, life-threatening genetic disorder.
0:35.5	After three doses, the baby's health significantly improved. So how does this gene-ed-ed-ed-treatening genetic disorder. After three doses, the baby's health significantly improved.
0:39.7	So how does this gene editing treatment work? What are the risks? And what does this
0:44.8	breakthrough mean for the 30 million people in the U.S. who have a rare genetic disease with no
0:50.1	available treatments? To help get some answers, I'm joined by the physician scientists who led
0:55.3	this research. Dr. Kieran Musunuru, Professor of Translational Research at the University of
1:00.6	Pennsylvania, and Dr. Rebecca Arons-Nichlis, Assistant Professor of Pediatrics and Genetics
1:05.5	at the Children's Hospital of Philadelphia and the University of Pennsylvania. Welcome to you
1:09.9	both to Science Friday.
1:16.1	Thank you so much for having us. Yeah, thanks for having us. Rebecca, let's start with your patient,
1:22.8	KJ. Tell us a little bit about him and how he became the first baby to get this treatment that you all developed. Yeah, so KJ was born last summer, and unfortunately, within the first two days of
1:30.1	life, he became quite sick. He had a very, very high level of a chemical in his blood called ammonia.
1:37.1	And so the ammonia was causing KJ to be very sleepy and not feed very well. And this is a huge medical
1:42.3	emergency because unless you can get rid of that ammonia,
1:45.9	the baby is at risk of having permanent brain injury. And unfortunately, a high number of infants
1:52.8	that are born with this type of disease die very early on in the neonatal period. And do we know
	...

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