21-Jul-2025
Prime editing treats childhood brain disease in mice
Broad Institute of MIT and HarvardPeer-Reviewed Publication
Researchers at the Broad Institute and The Jackson Laboratory have used prime editing, a precise and versatile form of gene editing, to correct the root cause of alternating hemiplegia of childhood (AHC), a debilitating genetic condition, in mice. The team used a scalable approach to develop prime editing treatments that directly repaired five different AHC-causing genetic mutations. Mice that received the treatment had far fewer and less severe symptoms of AHC, and survived more than twice as long as untreated mice.
- Journal
- Cell
- Funder
- NIH/National Institutes of Health, Chan Zuckerberg Initiative, RARE Hope, Alternating Hemiplegia of Childhood Foundation, U.S. National Science Foundation