Future perspective of induced pluripotent stem cells for diagnosis, drug screening and treatment of human diseases

Qizhou Lian; Yenyen Chow; Miguel Angel Esteban; Duanqing Pei; Hung-Fat Tse

doi:10.1160/TH10-05-0269

Thrombosis and Haemostasis, Table of Contents

Thromb Haemost 2010; 104(01): 39-44
DOI: 10.1160/TH10-05-0269

Theme Issue Article

Schattauer GmbH

Future perspective of induced pluripotent stem cells for diagnosis, drug screening and treatment of human diseases

Qizhou Lian

¹Cardiology Division, Department of Medicine, University of Hong Kong, Hong Kong, China

²Research Centre of Heart, Brain, Hormone, and Healthy Aging, University of Hong Kong, Hong Kong, Hong Kong, China

,

Yenyen Chow

¹Cardiology Division, Department of Medicine, University of Hong Kong, Hong Kong, China

²Research Centre of Heart, Brain, Hormone, and Healthy Aging, University of Hong Kong, Hong Kong, Hong Kong, China

,

Miguel Angel Esteban

³Stem Cell and Cancer Biology Group, Key Laboratory of Regenerative Biology, South China Institute for Stem Cell Biology and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, China

,

Duanqing Pei

³Stem Cell and Cancer Biology Group, Key Laboratory of Regenerative Biology, South China Institute for Stem Cell Biology and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences, Guangzhou, China

,

Hung-Fat Tse

¹Cardiology Division, Department of Medicine, University of Hong Kong, Hong Kong, China

› Author Affiliations

Abstract

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Summary

Recent advances in stem cell biology have transformed the understanding of cell physiology and developmental biology such that it can now play a more prominent role in the clinical application of stem cell and regenerative medicine. Success in the generation of human induced pluripotent stem cells (iPS) as well as related emerging technology on the iPS platform provide great promise in the development of regenerative medicine. Human iPS cells show almost identical properties to human embryonic stem cells (ESC) in pluripotency, but avoid many of their limitations of use. In addition, investigations into reprogramming of somatic cells to pluripotent stem cells facilitate a deeper understanding of human stem cell biology. The iPS cell technology has offered a unique platform for studying the pathogenesis of human disease, pharmacological and toxicological testing, and cell-based therapy. Nevertheless, significant challenges remain to be overcome before the promise of human iPS cell technology can be realised.

Keywords

Human induced pluripotent stem cells - reprogramming - in vitro disease models - drug screening

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References

References
1 Thomson JA, Itskovitz-Eldor J, Shapiro SS. et al. Embryonic stem cell lines derived from human blastocysts. Science 1998; 282: 1145-1147.
2 Rideout 3rd WM , Hochedlinger K, Kyba M. et al. Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy. Cell 2002; 109: 17-27.
3 Siu CW, Moore JC, Li RA. Human embryonic stem cell-derived cardiomyocytes for heart therapies. Cardiovasc Hematol Disord Drug Targets 2007; 07: 145-152.
4 Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 2006; 126: 663-676.
5 Takahashi K, Tanabe K, Ohnuki M. et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 2007; 131: 861-872.
6 Park IH, Zhao R, West JA. et al. Reprogramming of human somatic cells to pluripotency with defined factors. Nature 2008; 451: 141-146.
7 Yu J, Vodyanik MA, Smuga-Otto K. et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007; 318: 1917-1920.
8 Maherali N, Sridharan R, Xie W. et al. Directly reprogrammed fibroblasts show global epigenetic remodeling and widespread tissue contribution. Cell Stem Cell 2007; 02: 55-70.
9 Zhao XY, Li W, Lv Z. et al. iPS cells produce viable mice through tetraploid complementation. Nature 2009; 461: 86-90.
10 Chin MH, Mason MJ, Xie W. et al. Induced pluripotent stem cells and embryonic stem cells are distinguished by gene expression signatures. Cell Stem Cell 2009; 05: 111-123.
11 Maherali N, Hochedlinger K. Guidelines and techniques for the generation of induced pluripotent stem cells. Cell Stem Cell 2008; 03: 595-605.
12 Kiskinis E, Eggan K. Progress toward the clinical application of patient-specific pluripotent stem cells. J Clin Invest 2010; 120: 51-59.
13 Saha K, Jaenisch R. Technical challenges in using human induced pluripotent stem cells to model disease. Cell Stem Cell 2009; 05: 584-595.
14 Soldner F, Hockemeyer D, Beard C. et al. Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors. Cell 2009; 136: 964-977.
15 Colman A, Dreesen O. Pluripotent stem cells and disease modeling. Cell Stem Cell 2009; 05: 244-247.
16 Ebert AD, Svendsen CN. Human stem cells and drug screening: opportunities and challenges. Nat Rev Drug Discov 2010; 09: 367-372.
17 Ebert AD, Yu J, Rose Jr FF . et al. Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 2009; 457: 277-280.
18 Lee G, Papapetrou EP, Kim H. et al. Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs. Nature 2009; 461: 402-406.
19 Agarwal S, Loh YH, McLoughlin EM. et al. Telomere elongation in induced pluripotent stem cells from dyskeratosis congenita patients. Nature 2010; 464: 292-296.
20 Klivenyi P, Ferrante RJ, Matthews RT. et al. Neuroprotective effects of creatine in a transgenic animal model of amyotrophic lateral sclerosis. Nat Med 1999; 05: 347-350.
21 Shefner JM, Cudkowicz ME, Schoenfeld D. et al. NEALS Consortium. A clinical trial of creatine in ALS. Neurology. 2004: 631656-631661.
22 Rubin LL. Stem cells and drug discovery: the beginning of a new era?. Cell 2008; 132: 549-552.
23 Zhang J, Wilson GF, Soerens AG. et al. Functional cardiomyocytes derived from human induced pluripotent stem cells. Circ Res 2009; 104: e30-41.
24 Shiba Y, Hauch KD, Laflamme MA. Cardiac applications for human pluripotent stem cells. Curr Pharm Des 2009; 15: 2791-2806.
25 Tanaka T, Tohyama S, Murata M. et al. In vitro pharmacologic testing using human induced pluripotent stem cell-derived cardiomyocytes. Biochem Biophys Res Commun 2009; 385: 497-502.
26 Yokoo N, Baba S, Kaichi S. et al. The effects of cardioactive drugs on cardiomyocytes derived from human induced pluripotent stem cells. Biochem Biophys Res Commun 2009; 387: 482-488.
27 Siu CW, Liao SY, Liu Y. et al. Stem cells for myocardial repair. Thromb Haemost 2010; 104: 6-12.
28 Lawall H, Bramlage P, Amann B. Stem cell and progenitor cell therapy in peripheral artery disease. A critical appraisal. Thromb Haemost 2010; 103: 696-709.
29 Charwat S, Lang I, Dettke M. et al. Effect of intramyocardial delivery of autologous bone marrow mononuclear stem cells on the regional myocardial perfusion. NOGA-guided subanalysis of the MYSTAR prospective randomised study. Thromb Haemost 2010; 103: 564-571.
30 Murry CE, Keller G. Differentiation of embryonic stem cells to clinically relevant populations: lessons from embryonic development. Cell 2008; 132: 661-680.
31 Dimos JT, Rodolfa KT, Niakan KK. et al. Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons. Science 2008; 321: 1218-1221.
32 Si-Tayeb K, Noto FK, Nagaoka M. et al. Highly efficient generation of human hepatocyte-like cells from induced pluripotent stem cells. Hepatology 2010; 51: 297-305.
33 Song Z, Cai J, Liu Y. et al. Efficient generation of hepatocyte-like cells from human induced pluripotent stem cells. Cell Res 2009; 19: 1233-1242.
34 Zhang D, Jiang W, Liu M. et al. Highly efficient differentiation of human ES cells and iPS cells into mature pancreatic insulin-producing cells. Cell Res 2009; 19: 429-438.
35 Xu D, Alipio Z, Fink LM. et al. Phenotypic correction of murine hemophilia A using an iPS cell-based therapy. Proc Natl Acad Sci USA 2009; 106: 808-813.
36 Hanna J, Wernig M, Markoulaki S. et al. Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 2007; 318: 1920-1923.
37 Ye L, Chang JC, Lin C. et al. Induced pluripotent stem cells offer new approach to therapy in thalassemia and sickle cell anemia and option in prenatal diagnosis in genetic diseases. Proc Natl Acad Sci USA 2009; 106: 9826-9830.
38 Carr AJ, Vugler AA, Hikita ST. et al. Protective effects of human iPS-derived retinal pigment epithelium cell transplantation in the retinal dystrophic rat. PLoS One 2009; 04: e8152.
39 Nelson TJ, Martinez-Fernandez A, Yamada S. et al. Repair of acute myocardial infarction by human stemness factors induced pluripotent stem cells. Circulation 2009; 120: 408-416.
40 Zwi L, Caspi O, Arbel G. et al. Cardiomyocyte differentiation of human induced pluripotent stem cells. Circulation 2009; 120: 1513-1523.
41 Lian Q, Zhang Y, Zhang J. et al. Functional mesenchymal stem cells derived from human induced pluripotent stem cells attenuate limb ischemia in mice. Circulation 2010; 121: 1113-1123.
42 Hong H, Takahashi K, Ichisaka T. et al. Suppression of induced pluripotent stem cell generation by the p53-p21 pathway. Nature 2009; 460: 1132-1135.
43 Utikal J, Polo JM, Stadtfeld M. et al. Immortalization eliminates a roadblock during cellular reprogramming into iPS cell. Nature 2009; 460: 1145-1148.
44 Esteban MA, Wang T, Qin B. et al. Vitamin C enhances the generation of mouse and human induced pluripotent stem cells. Cell Stem Cell 2010; 06: 71-79.
45 Yamanaka S. A fresh look at iPS cells. Cell 2009; 137: 13-17.
46 Lee AS, Tang C, Cao F. et al. Effects of cell number on teratoma formation by human embryonic stem cells. Cell Cycle 2009; 08: 2608-2612.