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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

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. Vallabh soon found out that she inherited the same disease-causing mutation in the prion protein gene. Credit: Neumann EN, Bertozzi TM, et al.

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