Science Friday How Scientists Made The First Gene-Editing Treatment For A Baby
Jun 25, 2025
Dr. Kiran Musunuru, a leader in translational research, and Dr. Rebecca Ahrens-Nicklas, a pediatrician focused on genetics, join the discussion about a groundbreaking gene-editing treatment for a baby with a life-threatening disorder. They explain how CRISPR technology was tailored specifically for this case and share the emotional journey of the family involved. The conversation also touches on the scalability of such treatments, funding challenges, and the critical role of responsible media coverage in shaping public understanding of this medical advancement.
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KJ's Life-Threatening Disorder
- KJ was born with CPS1 deficiency, a rare urea cycle disorder causing dangerous ammonia buildup.
- Within days, he became very sick, risking permanent brain injury or death without treatment.
Gene Editing vs. Transplant
- Personalized CRISPR gene editing can target unique genetic variants causing diseases like KJ's.
- Liver transplant is risky, and gene editing offers a precise alternative to replace missing enzymes.
Parents Embrace Uncertain Treatment
- KJ's parents were told the risks and uncertainties candidly, including potential harm or no benefit.
- They supported the research to help their son and advance science despite the unknowns.