May 2025
1 dated step on the record this month, newest first.
- In-body gene editing
Doctors at Children's Hospital of Philadelphia and Penn Medicine treated a baby, KJ, born with a rare metabolic disease, with a gene-editing therapy made just for him and delivered to his liver in lipid nanoparticles. It was given safely, and he is growing well. The case was published in the New England Journal of Medicine.
Real-world testing