The prion protein is made from the PRNP gene. Editing approaches try to reduce how much of it a cell makes, either by switching the gene off with chemical tags (epigenetic editing) or by changing a letter of its code (base editing). The hope is a one-time treatment. Work so far is led by the Broad Institute and the Whitehead Institute, using a virus engineered to reach the brain, developed in the lab of Ben Deverman.
In mice, a single injection of the epigenetic editor CHARM (Science, June 2024, first author Edwin Neumann) removed more than 80 percent of brain prion protein. A base editor that installs a naturally occurring protective change (Nature Medicine, January 2025) cut prion protein by about half in mice carrying the human gene and extended their lifespan by about 50 percent. The authors of both describe the work as early and preclinical. Zinc finger repressors from Sangamo are listed by the CJD Foundation as preclinical and not enrolling. For drugs already in human trials, see PrP-lowering therapies.