Reinventing SOD1 Targeting with CRISPR-Based Gene Editing Technology to Diminish Mutant SOD1 & Restore Motor Neuronal Function

Time: 1:40 pm
day: Day Two Track A PM


  • Demonstrating disruption to the human SOD1 transgene using in vivo gene editing technology
  • Revealing functional restoration of motor neurones following intracerebroventricular injection
  • Utilizing this CRISPR editing preclinical approach for target evaluation