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Improvements in building in vitro pluripotent stem cell-derived disease models

Speaker
  • Staff Scientist, Thermo Fisher Scientific
    Biography
      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.

    Abstract

    The discovery of the CRISPR/Cas9 system has transformed research and now allows straightforward alterations in the genome and can be exploited to introduce changes to mimic diseases. This presentation will highlight the use of CRISPR/Cas9 in the generation of disease models in that are relevant for Parkinson’s disease. A review of our ongoing work on the development of pluripotent stem cell (PSC)-based disease models in the neuronal contexts covers which tools can be used to perform CRISPR/Cas9 based genome editing in PSC, how clonal isolation of edited PSC can be dramatically improved, how edited cells are characterized and how differentiation into the relevant cell type is used to reveal cellular phenotypes caused by the genetic changes.


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