Optimizing Animal Models for Genetic Diseases and Gene Editing Therapeutics

Time: 1:10 pm
day: Day One


  • Improving the sophistication of animal models using a combined approach of embryonic stem cells and CRISPR technology
  • Defining the unique requirements for animal models in the preclinical setting compared with basic academic research
  • Beyond proof-of-concept studies, exploring animal models as a tool to investigate unwanted safety issues of a therapy