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NOV 09, 2017 10:00 AM PST

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

Speaker

Abstract

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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NOV 09, 2017 10:00 AM PST

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


Specialty

Cell Biology

Biotechnology

Molecular Biology

Genetics

Cell Culture

Cancer Research

Biomarkers

Biochemistry

Gene Expression

Immunology

Genomics

Stem Cell Technologies

Dna

Gene Sequencing

Clinical Research

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