Human cerebral organoids as a therapeutic drug screening model for Creutzfeldt-Jakob disease.

ABSTRACT: Creutzfeldt-Jakob Disease (CJD) is a fatal, currently incurable, neurodegenerative disease. The search for candidate treatments would be greatly facilitated by the availability of human cell-based models of prion disease. Recently, an induced pluripotent stem cell derived human cerebral organoid model was shown to take up and propagate human CJD prions. This model offers new opportunities to screen drug candidates for the treatment of human prion diseases in an entirely human genetic background. Here we provide the first evidence that human cerebral organoids can be a viable model for CJD drug screening by using an established anti-prion compound, pentosan polysulfate (PPS). PPS delayed prion propagation in a prophylactic-like treatment paradigm and also alleviated propagation when applied

SUBMITTER: Groveman BR 

PROVIDER: S-EPMC7943797 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature