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Genetics:

CurePSP Research Roadmap. Scientific Hypotheses. Genetics: W ork out the cellular pathways controlled by the top 10 genes from CurePSP’s 2011 whole genome marker analysis and its 2014 whole exome sequencing project. Prions:

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Genetics:

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  1. CurePSP Research Roadmap Scientific Hypotheses Genetics: Work out the cellular pathways controlled by the top 10 genes from CurePSP’s 2011 whole genome marker analysis and its 2014 whole exome sequencing project. Prions: Work out steps in tau misfolding, templating and cell-to-cell transmission Proteins: Identify defects in production, handling and disposal of tau and other proteins in PSP/CBD Procedures Identify top 30-50 drug targets Perform high-throughput screens in molecular or cellular models Test drugs in vertebrate preclinical models New Experimental Tools Models: Create new cellular and animal lab models more faithful to human PSP/CBD Early-phase clinical trials by industry or academia Markers: Devise better tests for diagnosis and measurement of PSP/CBD and its treatment response in humans Late-phase clinical trials by industry

  2. CurePSP Research Roadmap • A general guide to the research topics that CurePSP sees as the path to PSP/CBD prevention • Uses the CurePSP-sponsored whole-genome SNP and WES results as a starting point for drug development. • Provides perspective/overview for potential donors. • Does not prohibit funding grants on other topics. • Are any updates/additions/deletions needed?

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