A therapy candidate for fatal prion diseases turns off disease-causing gene
Broad Institute
JUNE 27, 2024
By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. CRISPRoff uses building blocks from CRISPR gene editing technology, including the guide protein Cas9 that directs the tool to the target gene.
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