#5 Model systems & preclinical trials

Working Group 5
WG Name Model systems & preclinical trials
WG Leads Bernard Brais (Montréal, Canada)
Francesca Maltecca (Milan, Italy)
Hélène Puccio (Paris, France)
WG Objectives Establishing standards for aggregating digital-motor (sensors, video, audio, etc.) datasets in genetic ataxias, harmonized by cross-center standardized assessment protocols, for identification of digital-motor biomarkers in SCAs and ARCAs. This includes, but is not limited to

  • Sharing specific protocols and expertise for mouse phenotyping (behaviour and histology), neuronal cultures, imaging, biochemistry.
  • Sharing guidelines for preclinical trials (drug administration, outcome measures, statistics).
  • Sharing large data (gene expression, proteomics: experience, protocols and datasets).
  • Establishing peer support network (we plan to focus at first on developing a network around existing projects, to share our science and to identify common pathways to target. We will then solicit the joining of key model basic scientists from the ataxia field).
  • Creating an inventory of available human fibroblasts and/or iPSCs of ARCAs/SCAs (and other human material, i.e. blood, CSF, iPSCs).
  • Organizing Research lab meeting 1 every 2-4 months starting early 2021 in close collaboration with network of trainees.
  • Inserting a basic science session organized by the Models Work group for the next ARCA and SCA Global meeting.