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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. Previous research has shown that as little as 21 percent elimination of the protein can improve symptoms. Credit: Neumann EN, Bertozzi TM, et al.

Disease 142
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Rapid delivery of toxicological material

Drug Target Review

Advances in new technologies such as engineer transposase-based semi-targeted transgene integration, which integrates the genes of interest into highly active regions of the host genome, have enabled the generation of consistently and predictably high-yielding cell lines, as well as, supporting rapid stable pool generation.