iPSCs have the potential to treat a wide range of intractable diseases. In recent years, human embryonic stem cells (hESCs) have gained popularity as a potentially ideal cell candidate for regenerative medicine. hESCs are derived from the inner cell mass of the blastocyte and can be kept in an undifferentiated, self-renewing state indefinitely. In contrast to adult somafic cells, hESCs have the advantage of being pluripotent, which endows them with the ability to differentiate into virtually every cell type in the human body. However, the clinical use of human embryos is controversial in the US, and the problem of tissue rejection following transplantation in patients remains difficult. One way to circumvent these issues is to generate autologous iPSCs. Successful reprogramming of adult fibroblast cells into iPSCs based on defined factors was reported independently in 2008 by Shinya Yamanaka at Kyoto University, Japan {Oct4, Sox2, Klf4, c-Myc) and James Thomson at the University of Wisconsin (Ocf4, Sox2, Nanog, Lin28) (2). The main advantage of iPSCs is that they eliminate the need for human embryos or oocytes to generate patient-specific stem, cells and therefore can potentially bypass the ethical and political debates that have traditionally limited support for this field. A second important advantage is that the use of IPSCs obviates the need for immunosuppressive therapy because the cells are patient-specific. With the rapid progress in the iPSC field, patient-specific and disease-specific iPSCs from individuals with a variety of genetic diseases, such as Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson's disease (PD), amyotrophic lateral sclerosis (ALS), and Huntington disease (HD) have been generated(3,4). Furthermore, different derivatives and cell types have also been generated from IPSCs such as cardiomyocytes and motor neurons (4,5), making iPSCs an attractive candidate for regenerative medicine.

Agency
National Institute of Health (NIH)
Type
Research Program Projects (P01)
Project #
5P01GM099130-04
Application #
8730682
Study Section
Special Emphasis Panel (ZGM1)
Project Start
Project End
Budget Start
Budget End
Support Year
4
Fiscal Year
2014
Total Cost
Indirect Cost
Name
Stanford University
Department
Type
DUNS #
City
Stanford
State
CA
Country
United States
Zip Code
94304
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Matsa, Elena; Burridge, Paul W; Yu, Kun-Hsing et al. (2016) Transcriptome Profiling of Patient-Specific Human iPSC-Cardiomyocytes Predicts Individual Drug Safety and Efficacy Responses In Vitro. Cell Stem Cell 19:311-25
Burridge, Paul W; Diecke, Sebastian; Matsa, Elena et al. (2016) Modeling Cardiovascular Diseases with Patient-Specific Human Pluripotent Stem Cell-Derived Cardiomyocytes. Methods Mol Biol 1353:119-30
Hu, Shijun; Zhao, Ming-Tao; Jahanbani, Fereshteh et al. (2016) Effects of cellular origin on differentiation of human induced pluripotent stem cell-derived endothelial cells. JCI Insight 1:
Kodo, Kazuki; Ong, Sang-Ging; Jahanbani, Fereshteh et al. (2016) iPSC-derived cardiomyocytes reveal abnormal TGF-β signalling in left ventricular non-compaction cardiomyopathy. Nat Cell Biol 18:1031-42
Zhang, Xiaoyan; Marjani, Sadie L; Hu, Zhaoyang et al. (2016) Single-Cell Sequencing for Precise Cancer Research: Progress and Prospects. Cancer Res 76:1305-12
Liu, Renjing; Kim, Kun-Yong; Jung, Yong-Wook et al. (2016) Dnmt1 regulates the myogenic lineage specification of muscle stem cells. Sci Rep 6:35355
He, Chunjiang; Hu, Hanyang; Wilson, Kitchener D et al. (2016) Systematic Characterization of Long Noncoding RNAs Reveals the Contrasting Coordination of Cis- and Trans-Molecular Regulation in Human Fetal and Adult Hearts. Circ Cardiovasc Genet 9:110-8
Greer, Celeste B; Tanaka, Yoshiaki; Kim, Yoon Jung et al. (2015) Histone Deacetylases Positively Regulate Transcription through the Elongation Machinery. Cell Rep 13:1444-55
Ong, Sang-Ging; Lee, Won Hee; Kodo, Kazuki et al. (2015) MicroRNA-mediated regulation of differentiation and trans-differentiation in stem cells. Adv Drug Deliv Rev 88:3-15

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