Method

Teamed up with Vanderbilt University cardiologist Jonathan Brown and Francis Collins, director of the National Institutes of Health, whose group was one of two that identified the progeria mutation in 2003. The team first tested the base-editing approach in cultured cells from two progeria patients, finding that it corrected the mutation while making few unwanted changes elsewhere in the genome. They then packaged DNA encoding the base editor into adeno-associated viruses (AAVs), a standard delivery vehicle for gene therapies, and injected these into young mice with the progeria mutation.