Uploaded August 2025 | Updated September 2026, 2 weeks ago
In May, 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, assistant professor of pediatrics and genetics at the Children’s Hospital of Philadelphia and the University of Pennsylvania
Dr. Kiran Musunuru, professor of translational research at the University of Pennsylvania
Subscribe to our podcast and never miss your daily dose of science: pod.link/73329284
In May, 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, assistant professor of pediatrics and genetics at the Children’s Hospital of Philadelphia and the University of Pennsylvania
Dr. Kiran Musunuru, professor of translational research at the University of Pennsylvania
Subscribe to our podcast and never miss your daily dose of science: pod.link/73329284
