Novel Humanized Mouse Models of ALS Recapitulate the Human Molecular & Pathological Hallmarks of ALS But Overcome the Artifacts Associated with TDP-43 Over-Expression

Time: 10:45 am
day: Day Two Track A AM


  • Overexpression of TDP-43 leads to aggressive phenotypes and potential artifacts that are not related to ALS
  • Humanized C9orf72 and TDP-43 mouse models show degeneration, TDP-43 mislocalization, aggregation and mis-splicing of TDP-43 targets
  • These models can be used to rapidly screen therapeutics that restore TDP-43 function