Transforming Gene Therapy: How Induced Pluripotent Stem Cells Facilitate AAV Testing

Gene therapy, best defined as administering nucleic acids to treat, cure, or prevent disorders, holds enormous potential in the field of medicine. The growing success of these therapies has prompted an increasing need for alternative testing platforms for thorough investigation of the pathophysiology of the target disease. Furthermore, accurate models need to be established to provide the proper benchmark for testing the efficacy of each therapeutic approach as well as to give insight to any adverse side effects.

Human iPSC models with their close fidelity to human physiology can fulfil both roles. Their ability to recapitulate main disease phenotypes provides insights in disease pathophysiology and translational information on therapeutic effects.

In this webinar we will explore the value iPSCs can bring in the gene therapy development process, and how iPSC technology can enable researchers to accelerate their pre-clinical trial pipelines. A case study of a recently completed project will clearly demonstrate the positive contribution of iPSC models during the development of gene therapies.