#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.

If you are interested in participating in working group 5, please follow this link.