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A designer chaperone against prion diseases

A computationally designed small molecule inhibits the replication of prion-protein aggregates and ameliorates neurological symptoms in mouse and non-human-primate models of transmissible spongiform encephalopathy.

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Fig. 1: Experimental workflow for the identification and characterization of an anti-prion-protein pharmacological chaperone.

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Correspondence to Emiliano Biasini.

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Biasini, E. A designer chaperone against prion diseases. Nat Biomed Eng 3, 167–168 (2019). https://doi.org/10.1038/s41551-019-0367-6

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