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

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.

Guests: 
Dr. Rebecca Ahrens-Nicklas is an assistant professor of pediatrics and genetics at the Children's Hospital of Philadelphia and the University of Pennsylvania.
Dr. Kiran Musunuru is a professor of translational research at the University of Pennsylvania.

Transcripts for each episode are available within 1-3 days at sciencefriday.com.

Subscribe to this podcast. Follow our show on Instagram, TikTok, Facebook, and Bluesky @scifri and sign up for our newsletters. Got a science question that’s keeping you up at night? Call us: 877-472-4374

Hosted by Simplecast, an AdsWizz company. See pcm.adswizz.com for information about our collection and use of personal data for advertising.