A therapy candidate for fatal prion diseases turns off disease-causing gene
Broad Institute
JUNE 27, 2024
Unlike gene editing, this “epigenetic” editing does not modify the underlying DNA sequence, but it should switch the gene off permanently, which means that this could be a one-time treatment. At first, they used part of a methyltransferase, a molecule that adds methyl groups to DNA, called DNMT3A.
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