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AUG 30, 2016 8:00 AM PDT

Delivery solutions for robust CRISPR-based genomic editing in stem cells for disease model generation

Speaker
  • Staff Scientist, Cell Biology, Thermo Fisher Scientific
    Biography
      Nektaria Andronikou joined the research and development team at Thermo Fisher Scientific in 2010 working within the R&D transfection delivery team. She is currently involved in the development of delivery technologies targeting relevant cellular models and in vivo systems that will enable the use of exciting new technologies, such as CRISPR, messengerRNA and cell therapy. Nektaria began her professional career at ISIS Pharmaceuticals, as a research associate for the Cardiovascular Drug Discovery program, screening numerous pre-clinical targets that led to the discovery of the now FDA approved antisense drug, Kynamro. She received a Bachelor of Science in Biochemistry with a minor in Cellular and Molecular Biology from UCSD.

    Abstract
    Stem cells, specifically induced pluripotent stem cells (iPSCs), offer exciting potential for the future of cell therapy and regenerative medicine. More recently, genetic engineering of stem cells and progenitor lines is enabling in vitro disease modeling for drug development with cell populations that would otherwise be unavailable from live donors.  With the use of TALENs and CRISPRs, this true power is fast approaching.  Having the ability to develop two cell models with isogenic background, except for the site specific edit, gives researchers the potential to study the true effects of a single mutation in a pathway or syndrome.  However, the lack of advanced technologies has been hindering the current pace of research and discovery.  Improved delivery can help accelerate research for emerging new therapies and help the understanding of disease pathways and mechanisms.  We will review multiple delivery solutions for CRISPR based technologies and also highlight protocol details and data outcomes for an iPSC specific CRISPR experiment.

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