Gene-Editing Therapy for Prion Disease: A New Hope

May 4, 2025

Gene-editing therapy for prion disease has emerged as a groundbreaking potential solution for a class of rare and invariably fatal disorders caused by misfolded proteins in the brain.Recent research out of the Broad Institute of MIT and Harvard has shown promising results, revealing that modifying a single base in the prion protein gene can significantly reduce harmful protein levels in affected mice, extending their lifespans by an impressive 52%.
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