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Stem Cell Engineering and Differentiation for Disease Modeling and Cell-based Therapies

1 Department of Biomedical Engineering, The Pennsylvania State University, University Park, PA, 16802, USA
2 The Huck Institutes of the Life Sciences, The Pennsylvania State University, University Park, PA, 16802, USA
3 Department of Biology, The Pennsylvania State University, University Park, PA, 16802, USA
§ These authors contributed equally.

Special Issue: iPS Cell Technologies in Human Diseases

The identification and characterization of stem cells, especially human embryonic stem cells, has revolutionized the field of developmental biology by providing an in vitro system to study human development. In addition, reprogramming adult cells from patients into an embryonic stem cell-like state using induced pluripotent stem cell (iPSC) technology can potentially generate an unlimited source of human tissue carrying genetic mutations that caused or facilitated disease development, providing unprecedented possibilities to model human disease in the culture dish. To do this, however, efficient differentiation methods to direct iPSCs through multiple progenitor stages to yield homogeneous populations of somatic cells must be established. Furthermore, disease modeling using iPSCs requires proper controls for this “disease-in-a-dish” approach. Therefore, methods to efficiently engineer the genome of iPSCs to correct the mutations become vital in stem cell research. Here we reviewed the iPSC generation techniques and several genome-editing tools, such as TALENs and CRISPR-Cas9, for performing iPSC gene knock-in and knockout. We also present several efficient stem cell directed differentiation methods for converting iPSCs to neural, hematopoietic, cardiac, and pancreatic lineages. Together, this knowledge will provide insight into design principles for disease modeling using iPSCs and stem cell-based therapies.
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Copyright Info: © 2017, Xiaojun Lian, et al., licensee AIMS Press. This is an open access article distributed under the terms of the Creative Commons Attribution Licese (http://creativecommons.org/licenses/by/4.0)

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