A therapy candidate for fatal prion diseases turns off disease-causing gene
Broad Institute
JUNE 27, 2024
CRISPRoff silences the targeted gene by adding methyl groups, chemical tags that prevent the gene from being transcribed or read into RNA and so from being expressed as protein. The first problem they had to tackle was the editor’s size, because the editor needs to be small enough to be packaged and delivered to specific cells in the body.
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