Spiegazione semplice
Kiran Musunuru is a cardiologist who also runs a genetics laboratory. With colleagues at Children's Hospital of Philadelphia, he led the team that designed, made and administered a gene-editing treatment built for one infant's specific mutation — from diagnosis to treatment in about six months. He also co-founded the company developing a one-time treatment for high cholesterol.
Approfondisci
Kiran Musunuru is Professor of Cardiovascular Medicine and Genetics at the University of Pennsylvania. He co-founded Verve Therapeutics and led, with colleagues at Children's Hospital of Philadelphia, the development of a patient-specific in vivo base editor for severe CPS1 deficiency — the first personalised gene-editing medicine, published in the New England Journal of Medicine in 2025.
Sources
- New England Journal of Medicine (Musunuru et al.) · 2025
Patient-specific in vivo gene editing to treat a rare genetic disease ↗