Abstract
The breakthrough development of induced pluripotent stem cell (iPSC) technology is not only revolutionizing basic stem cell science but is also spurring efforts to reprogram one somatic cell type directly into another. Induced pluripotent stem cells provide scientists with a self-renewing and, thus, unlimited, source of pluripotent cells for targeted differentiation, in principle, into the entire range of cell types found in the body. Therefore, iPSC technology and the increasingly refined abilities to differentiate iPSCs into disease-relevant mature cells has far reaching implications for understanding disease etiology and promoting drug discovery and other advances in regenerative medicine. In this review, we summarize the latest progress in the application of patient-specific iPSCs for disease modeling, drug screening and cell replacement therapy, and discuss their impact on precision personalized medicine.
Highlights
The establishment of human embryonic stem cells in 1998 together with their unlimited self-renewal potential and ability to differentiate into any cell type of the body has raised hopes for drug discovery and regenerative medicine [1]
This changed dramatically in 2006 when Kazutoshi Takahashi and Shinya Yamanaka made the seminal discovery that mouse skin fibroblasts can be reprogrammed into an induced pluripotent stem cell state that shares the indefinite self-renewal and pluripotent differentiation capacities of ESCs using a simple cocktail of pluripotency transcription factors [2]
These same investigators as well as groups headed by James Thomson and George Daley succeeded in converting human fibroblasts into hiPSCs [3,4,5]
Summary
The establishment of human embryonic stem cells (hESCs) in 1998 together with their unlimited self-renewal potential and ability to differentiate into any cell type of the body has raised hopes for drug discovery and regenerative medicine [1]. The challenges related to bioethics, safety and the limited availability of diseasespecific hESC lines have complicated the realization of these hopes This changed dramatically in 2006 when Kazutoshi Takahashi and Shinya Yamanaka made the seminal discovery that mouse skin fibroblasts can be reprogrammed into an induced pluripotent stem cell (iPSC) state that shares the indefinite self-renewal and pluripotent differentiation capacities of ESCs using a simple cocktail of pluripotency transcription factors [2]. One year later, these same investigators as well as groups headed by James Thomson and George Daley succeeded in converting human fibroblasts into hiPSCs [3,4,5]. Taken together these advances enable the generation of patientand disease-specific hiPSCs as avenues for exploring disease etiology, developing novel drugs, toxicology screening and, in the future, cell replacement therapies
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