Director of R&D, Cell Biology, Thermo Fisher Scientific
BIOGRAPHY
MAY 01, 2017 8:00 AM PDT
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Using iPSCs and Genome Engineering to Build Disease Models
Presented at:
4th Annual 24 Hours of Stem Cells™ virtual event
Speaker
Abstract
Developing therapies 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 cell models for these in vitro approaches. We review current advances in genome engineering and how to leverage a portfolio of these tools to generate knock-out and knock-in models for use in target or compound identification. Specifically, we demonstrate this approach with induced pluripotent stem cells (iPSCs) to build isogenic disease models, which can be further differentiated to various cell types of interest that 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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MAY 01, 2017 8:00 AM PDT
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Using iPSCs and Genome Engineering to Build Disease Models
Presented at:
4th Annual 24 Hours of Stem Cells™ virtual event
Specialty
Molecular Biology
Biotechnology
Cell Biology
Cell Culture
Neuroscience
Genetics
Cancer Research
Gene Expression
Crispr-Cas9
Clinical Research
Dna
Immunology
Pcr/rt-Pcr/real-Time Pcr
Biomarkers
Gene Editing
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Africa1%
Registration Source
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Biologist5%
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Biotech Company4%
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