New Epigenetic Tool Shows Promise for Treating Fatal Prion Diseases

Researchers at MIT and Harvard develop CHARMs to silence harmful genes, potentially offering a one-time treatment for neurodegenerative disorders.

CHARMs target prion protein genes to prevent production of misfolded proteins causing neurodegeneration.
The tool uses zinc finger proteins for precise gene silencing and minimal off-target effects.
Initial tests in mice show an 80% reduction in prion proteins, significantly above the required threshold to improve symptoms.
The collaboration between Broad and Whitehead Institutes has fast-tracked the development of this potential therapy.
Further research and trials are needed to confirm safety and efficacy in humans.