Preclinical Modeling – What Advocates Need to Know about Animal & Cell Models

June 24, 2021

*Recommened for Patients/Advocates


Advances in Animal Models

  • How have approaches to animal models evolved in rare disease research and explore what advocates need to know to evaluate animal models in their own disease state/community?
  • What are animal models used for and what are the most common animal models?
  • What are new animal models that are being explored and showing potential in rare disease and beyond?
  • What do you need to know before you start looking for an animal model?
  • How do you evaluate the right animal model for your disease state?
  • How long will it take to develop an animal model and how much will it cost?

Understanding Induced Pluripotent Stem Cells (iPSC) in Disease Modeling

  • Learn what patients and advocates need to know to evaluate using iPSCs in their disease state/research activities Explore iPSCs role in preclinical research and where they can reduce reliance animal models
  • Discuss how iPSCs can be a tool to de-risk and validate research for clinical studies
  • Define iPSC cell lines and their role in preclinical research o Review timelines and costs related to developing and validating iPSCs
  • Explore opportunities for advocates to utilize iPSCs in their early research

Pitfalls in Animal Models: What Do You Do When Your Mouse Model Doesn’t Work/Case Study


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