Notes from March 3, 2012 – Day 3 of the prion workshop at Koch Institute

The final day of the workshop consisted mostly of setting goals and prioritizes and identifying people and resources to get things done.  Some of the key immediate goals are:

  • Compilation of data from available clinical reports on human FFI
  • Gathering of more detailed patient metadata and, where possible, whole genomes, through outreach.  The purpose of both this and the above bullet are to better understand the variation in natural history of FFI across patients and families and determine whether there are manipulable associated factors.
  • Convergence on a set of variables, agents and pathways to test in mouse models
  • Development of human FFI stem cells for therapeutic testing
  • Develop specific aims for grant writing
  • Find ways to keep more FFI mice and improve mouse model
  • Identify parameters for potential assay