Eenvoudige uitleg
This is the hospital that, with the University of Pennsylvania, designed and gave a gene-editing treatment built for one baby's specific mutation. It is also a long-standing centre for gene therapy in children.
Dieper ingaan
Children's Hospital of Philadelphia, with the University of Pennsylvania, developed and administered the first patient-specific in vivo base-editing therapy, for an infant with severe CPS1 deficiency, in 2025. It has a long history in paediatric gene therapy including the development pathway of voretigene neparvovec for inherited retinal dystrophy.
Sources
- New England Journal of Medicine · 2025
Patient-specific in vivo gene editing to treat a rare genetic disease ↗