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Research Institute, report the results of preclinical studies of an antisense therapy against prion disease. In this new work, using an expanded set of prion protein -targeting antisense oligonucleotides, the authors have laid the basis for full scale clinical development. This research shows that, across multiple treatment paradigms, reducing levels of prion protein in prion-infected lab animals significantly extends their survival.
Researchers here showed that reducing levels of prion protein can triple the survival of prion-infected animals. Even reducing prion protein levels by a small amount, which should be easier to achieve clinically, resulted in significant survival benefits.
Reduction of prion protein is effective across prion strains and across a battery of different treatment timepoints. The researchers show that reducing prion protein is effective before any symptoms are seen. They also demonstrate, for the first time, that a single dose of a prion protein -lowering treatment can reverse markers of disease even after toxic clumps have formed in the brain.