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Advancing PSP/CBD Research: A Strategic Roadmap for Understanding and Treatment

This roadmap outlines the key research directions for understanding Progressive Supranuclear Palsy (PSP) and Corticobasal Degeneration (CBD). It focuses on identifying cellular pathways controlled by critical genes, unraveling tau protein misfolding mechanisms, and pinpointing drug targets. The roadmap emphasizes developing new experimental tools, models, and diagnostics to facilitate early and late-phase clinical trials. By leveraging insights from genetic analyses, this guide offers a comprehensive strategy for potential donors and researchers to contribute to PSP/CBD prevention and treatment advancements.

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Advancing PSP/CBD Research: A Strategic Roadmap for Understanding and Treatment

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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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