NOV 09, 2017 10:00 AM PST

Eliminating inherent genome editing bottlenecks in iPSCs to build physiologically relevant disease models

  • Staff Scientist, Thermo Fisher Scientific
      Dr. Erik Willems was trained as a stem cell biologist in Brussels, Belgium where he obtained his PhD in 2007, after which he soon relocated to San Diego, California to develop his expertise in the use of pluripotent stem cells in high throughput screening assays for understanding the basic biology and disease of the developing heart at the Sanford Burnham Prebys Medical Discovery Institute. Dr. Willems then pursued his passion for the development of biotechnology tools and applications and joined Thermo Fisher Scientific in Carlsbad, California where he - as a Manager in the Cell Biology group - currently leads a team focused on pluripotent stem cell-based customer driven projects and product applications, including characterization, reprogramming, genome editing, differentiation and disease modeling with a focus on drug discovery applications. Now in the stem cell field for over 15 years, Dr. Willems published numerous peer reviewed articles including in high impact journals such as Cell Stem Cell. His key expertise includes pluripotent stem cell biology, differentiation, genome editing, high throughput screening and drug discovery.


    Therapeutic development for human diseases continues to face obstacles, particularly in translating targets or compounds identified by in vitro screening campaigns to valid targets or efficacious and safe compounds once tested in humans.  Here we discuss strategies that leverage induced pluripotent stem cells (iPSCs) to increase the relevance of human cell models for these in vitro approaches.  We review current advances in genome engineering to pivot from challenges with delivery, identification and selection and subsequent clonal outgrowth by leveraging tools to consistently and reliably generate knock-out and knock-in models for use in target or compound identification.  Specifically, we demonstrate this approach with iPSCs to build isogenic disease models, which can be further differentiated to various cell types of interest such as cardiomyocytes and dopaminergic neurons to model disease and are more directly related to a disease area than commonly used immortalized cell lines.  We expect strategies combining genome engineering and stem cells to provide platforms for more robust disease models that will provide more predictable translation of in vitro to in vivo results.

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