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MIT and Harvard Develop CHARM, New Epigenetic Tool for Prion Diseases

Jun 27, 2024, 09:17 PM

Researchers from MIT and Harvard have developed a new epigenetic editing tool called CHARM, which shows promise as a therapy for prion diseases. This tool, created in collaboration with the Whitehead Institute and Broad Institute, can methylate DNA at the promoter of targeted genes, effectively silencing the prion protein gene in mice. The compact nature of CHARM allows it to fit into an adeno-associated virus (AAV), facilitating brain-wide delivery and gene silencing. Chroma Medicine has licensed CHARM for use beyond prion diseases, indicating its potential for broader applications in treating neurological disorders such as Parkinson’s and Alzheimer’s. This development, published in Science, represents a significant step towards gene therapy for diseases that currently lack effective treatments. The research was led by Jonathan Weissman and targets diseases like Creutzfeldt-Jakob.