Abstract
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.
Highlights
The contemporaneous generation of human induced pluripotent stem cells by the Yamanaka and Thomson groups has revolutionized the stem cell research field and provided new avenues of research, as well as renewed focus on the development of cell therapies and disease models [1,2]
The loss of cell function or cell death occurring in a cell type unable to self-regenerate presents a challenge in the development of therapeutics for degenerative diseases, such as Parkinson‟s, heart failure, or type II diabetes
This is due to the lack of a cell source for many difficult to obtain cells, such as neurons, cardiomyocytes, or beta cells, that are often the cells affected by loss of function or cell death in degenerative disease
Summary
The contemporaneous generation of human induced pluripotent stem cells (iPSCs) by the Yamanaka and Thomson groups has revolutionized the stem cell research field and provided new avenues of research, as well as renewed focus on the development of cell therapies and disease models [1,2]. The emergence of iPSC technology has provided a renewable stem cell source free of the controversy surrounding human embryonic stem cells (hESCs) and introduced the concept of cellular reprogramming, highlighting cell state plasticity. This technology has provided an unprecedented opportunity to study human development, model genetic diseases, and develop therapeutics for the treatment of a variety of disease conditions, in particular degenerative disorders. Cellular reprogramming and directed differentiation techniques are crucial to the development of cell-based therapies for degenerative diseases This is due to the lack of a cell source for many difficult to obtain cells, such as neurons, cardiomyocytes, or beta cells, that are often the cells affected by loss of function or cell death in degenerative disease.
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