Epigenetic Editor Silences Toxic Proteins in the Mouse Brain, Offering Promising Path to Treat Deadly Prion Diseases

Posted onJuly 25th, 2024byDr. Monica M. Bertagnolli

Prion diseases are fatal neurodegenerative disorders caused by a malfunction of the prion protein in the brain. Exposure to a misfolded version of the protein triggers normal proteins of the same type in the brain to misfold, forming clumps that produce infectious disease and fatal brain damage over time. There are currently no treatments, preventive vaccines, or cures for prion diseases, which can be acquired, like mad cow disease, or inherited, like fatal familial insomnia. But an encouraging new study in mice suggests a potentially promising path for developing a treatment for people with these deadly conditions.

Findings from an NIH-supported study reported inScienceshow that the key to this approach is a molecular tool capable of silencing prion protein throughout the brain using epigenetic editing. Unlike gene editing approaches, which change the sequence of genes, epigenetic editing can turn gene expression off with the addition of a chemical tag that prevents genes from being translated into proteins. Such a strategy may be able to deliver modifying tools to the brain or other parts of the body to silence specific toxic or disease-causing genes, including the one encoding the prion protein, without the risks associated with altering DNA sequences.