Design, Screening & Optimization of Oligonucleotide Therapeutics with Optimal Profiles Across Large-Scale Data Sets of Individual Neurons for Targeting CNS Diseases

Time: 3:00 pm
day: Pre-Conference Discussion Day


  • Exploring antisense oligonucleotide (ASO) design, screening and optimization
  • for CNS applications, with a focus on assessment of ASOs at the cellular level in diverse neuronal types
  • Outlining the development of deep-learning techniques with large scale data sets to characterize desired phenotype rescue and undesired toxicities associated with candidate ASO therapeutics
  • Revealing findings in the context of CNS disorders such as genetic epilepsies and chronic pain conditions