Gene editing extends lifespan in mouse model of prion disease
A single-letter edit in DNA reduces levels of the disease-causing prion protein in the brain and could lead to a preventative, one-time treatment for the deadly neurodegenerative disorder.

Credit: Maria Nemchuk, Broad Communications
Eric Minikel and Sonia Vallabh run a lab with a singular focus: preventing and treating prion disease within their lifetime.
Funding
This work was supported in part by the National Institutes of Health, Prion Alliance, and the Howard Hughes Medical Institute.
Paper cited
An M, Davis JR, et al. In vivo base editing extends lifespan of a humanized mouse model of prion disease. Nature Medicine. Online January 14, 2025. DOI: 10.1038/s41591-024-03466-w.