MIT and Harvard Develop CHARM, an Epigenetic Gene-Silencing Tool, to Combat Prion Diseases

Jun 27, 2024, 07:38 PM

Researchers from MIT and Harvard, in collaboration with the Broad Institute and Whitehead Institute, have developed a new, powerful gene-silencing tool called CHARM. This epigenetic editor shows promise as a therapy against fatal prion diseases by turning off the disease-causing gene. The compact epigenetic editor is small enough to fit into an adeno-associated virus (AAV), which allows it to permeate throughout the brain and effectively silence prions in mice. Chroma Medicine has licensed CHARM for use outside prion disease, paving the way for a new class of genetic approaches to treat certain diseases.