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Last month, scientists reported a historic first: they gave the first personalized gene-editing treatment to a baby who was born with a rare life-threatening genetic disorder. Before the treatment, his prognosis was grim. But after three doses, the baby’s health improved.
So how does it work? What are the risks? And what could this breakthrough mean for the 30 million people in the US who have a rare genetic disease with no available treatments?
To help get some answers, Host Flora Lichtman is joined by the physician-scientists who led this research: geneticist Dr. Kiran Musunuru and pediatrician Dr. Rebecca Ahrens-Nicklas.
Further Reading
- Read more about this story via the New York Times.
- Watch a video from the Children’s Hospital of Philadelphia to meet KJ and learn about his treatment.
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Segment Guests
Rebecca Ahrens-Nicklas
Dr. Rebecca Ahrens-Nicklas is an assistant professor of pediatrics and genetics at the Children’s Hospital of Philadelphia and the University of Pennsylvania.
Kiran Musunuru
Dr. Kiran Musunuru is a professor of translational research at the University of Pennsylvania.
Segment Transcript
The transcript is being processed. It will be available 2-3 days after this story’s publication date.
Meet the Producers and Host
About Dee Peterschmidt
Dee Peterschmidt is a producer, host of the podcast Universe of Art, and composes music for Science Friday’s podcasts. Their D&D character is a clumsy bard named Chip Chap Chopman.
About Flora Lichtman
Flora Lichtman is a host of Science Friday. In a previous life, she lived on a research ship where apertivi were served on the top deck, hoisted there via pulley by the ship’s chef.