Health Report - Separate stories podcast There's been a big CRISPR breakthrough. What does the future hold?
May 23, 2025
Marco Herold, CEO of the Olivia Newton-John Cancer Research Institute, delves into the groundbreaking world of CRISPR gene editing. He discusses the first-ever personalized gene therapy treatment for a baby, highlighting its transformative potential. The conversation covers innovative CRISPR applications in cancer treatment, including new methods enhancing immune responses against glioblastoma. Herold also shares insights on the rapid development timeline of these therapies and their future implications in medicine, emphasizing both the excitement and challenges ahead.
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CRISPR Base Editing Breakthrough
- CRISPR base editing allows precise swapping of a single wrong DNA letter back to the correct one.
- This technique was used to treat a baby with a rare liver enzyme deficiency by editing his DNA at the molecular level.
How CRISPR Base Editing Works
- CRISPR uses a Cas enzyme guided by RNA to find and edit a precise DNA address.
- Base editing disables full DNA cutting and instead chemically converts one DNA letter to another for precise mutation correction.
First Personalized CRISPR Therapy Story
- The edited DNA allowed the baby's liver to produce the necessary enzyme and prevent toxic ammonia buildup.
- The therapy used lipid nanoparticles, like those in COVID vaccines, to deliver CRISPR safely to liver cells.