Translational prospects for human induced pluripotent stem cells

Papers of special note have been highlighted as:▪ of interest ▪▪ of considerable interest

Bibliography

• 1  Thomson JA, Itskovitz-Eldor J, Shapiro SS et al.: Embryonic stem cell lines derived from human blastocysts. Science282(5391),1145–1147 (1998).
  Medline CASGoogle Scholar
• 2  Takahashi K, Yamanaka S: Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell126(4),663–676 (2006).▪▪ The original description of induced pluripotency came from Yamanaka in Kyoto, Japan, and this Kyoto group has continued to pioneer induced pluripotent stem (iPS) cell technology.
  Medline CASGoogle Scholar
• 3  Takahashi K, Tanabe K, Ohnuki M et al.: Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell131(5),861–872 (2007).
  Medline CASGoogle Scholar
• 4  Muller FJ, Laurent LC, Kostka D et al.: Regulatory networks define phenotypic classes of human stem cell lines. Nature455(7211),401–405 (2008).▪ A systems level understanding of similarities and differences between various pluripotent cell types will require the analysis of large, complex datasets. Here an expression-level signature for pluripotency is defined from an analysis of multiple pluripotent cell lines.
  MedlineGoogle Scholar
• 5  Marchetto MC, Yeo GW, Kainohana O, Marsala M, Gage FH, Muotri AR: Transcriptional signature and memory retention of human-induced pluripotent stem cells. PLoS One4(9),e7076 (2009).
  MedlineGoogle Scholar
• 6  Park I-H, Zhao R, West JA et al.: Reprogramming of human somatic cells to pluripotency with defined factors. Nature451(7175),141–146 (2008).
  Medline CASGoogle Scholar
• 7  Chin MH, Mason MJ, Xie W et al.: Induced pluripotent stem cells and embryonic stem cells are distinguished by gene expression signatures. Cell Stem Cell5(1),111–123 (2009).
  Medline CASGoogle Scholar
• 8  Boland MJ, Hazen JL, Nazor KL et al.: Adult mice generated from induced pluripotent stem cells. Nature461(7260),91–94 (2009).
  Medline CASGoogle Scholar
• 9  Zhao XY, Li W, Lv Z et al.: iPS cells produce viable mice through tetraploid complementation. Nature461(7260),86–90 (2009).
  Medline CASGoogle Scholar
• 10  Osafune K, Caron L, Borowiak M et al.: Marked differences in differentiation propensity among human embryonic stem cell lines. Nat. Biotechnol.26(3),313–315 (2008).
  Medline CASGoogle Scholar
• 11  Izpisua Belmonte JC, Ellis J, Hochedlinger K, Yamanaka S: Induced pluripotent stem cells and reprogramming: seeing the science through the hype. Nat. Rev. Genetics10(12),878–883 (2009).
  Medline CASGoogle Scholar
• 12  Yu J, Vodyanik MA, Smuga-Otto K et al.: Induced pluripotent stem cell lines derived from human somatic cells. Science318(5858),19177–11920, 2007.
  Google Scholar
• 13  Yoshida Y, Takahashi K, Okita K, Ichisaka T, Yamanaka S: Hypoxia enhances the generation of induced pluripotent stem cells. Cell Stem Cell5(3),237–241 (2009).
  Medline CASGoogle Scholar
• 14  Esteban MA, Wang T, Qin B et al.: Vitamin C enhances the generation of mouse and human induced pluripotent stem cells. Cell Stem Cell6(1),71–79 (2009).
  MedlineGoogle Scholar
• 15  Jaenisch R, Bird A: Epigenetic regulation of gene expression: how the genome integrates intrinsic and environmental signals. Nat. Genetics33S,245–254 (2003).
  Google Scholar
• 16  Pick M, Stelzer Y, Bar-Nur O, Mayshar Y, Eden A, Benvenisty N: Clone- and gene-specific aberrations of parental imprinting in human induced pluripotent stem cells. Stem Cells27(11),2686–2690 (2009).
  Medline CASGoogle Scholar
• 17  Deng J, Shoemaker R, Xie B et al.: Targeted bisulfite sequencing reveals changes in DNA methylation associated with nuclear reprogramming. Nat. Biotechnol.27(4),353–360 (2009).
  Medline CASGoogle Scholar
• 18  Ball MP, Li JB, Gao Y et al.: Targeted and genome-scale strategies reveal gene-body methylation signatures in human cells. Nat. Biotechnol.27(4),361–368 (2009).
  Medline CASGoogle Scholar
• 19  Doi A, Park I-H, Wen B et al.: Differential methylation of tissue- and cancer-specific CpG island shores distinguishes human induced pluripotent stem cells, embryonic stem cells and fibroblasts. Nat. Genetics41(12),1350–1353 (2009).
  Medline CASGoogle Scholar
• 20  Ram EV, Meshorer E: Transcriptional competence in pluripotency. Genes Dev.23(24),2793–2798 (2009).
  MedlineGoogle Scholar
• 21  Xu J, Watts JA, Pope SD et al.: Transcriptional competence and the active marking of tissue-specific enhancers by defined transcription factors in embryonic and induced pluripotent stem cells. Genes Dev.23(24),2824–2838 (2009).
  Medline CASGoogle Scholar
• 22  Zhao X, Ruan Y, Wei C-L: Tackling the epigenome in the pluripotent stem cells. J. Genet. Genomics35(7),403–412 (2008).
  Medline CASGoogle Scholar
• 23  Shih CC, Forman SJ, Chu P, Solvak M: Human embryonic stem cells are prone to generate primitive, undifferentiated tumors in engrafted human fetal tissues in severe combined immunodeficient mice. Stem Cells Dev.16(6),893–902 (2007).
  Medline CASGoogle Scholar
• 24  Miura K, Okada Y, Aoi T et al.: Variation in the safety of induced pluripotent stem cell lines. Nat. Biotechnol.27(8),743–745 (2009).▪▪ Neurospheres generated from iPS cells were transplanted and followed for teratoma potential. Teratoma propensity varied widely, dependent on the somatic cell origin of the iPS cell.
  Medline CASGoogle Scholar
• 25  Kazuki Y, Hiratsuka M, Takiguchi M et al.: Complete genetic correction of iPS cells from Duchenne muscular dystrophy. Mol. Ther.18(2),386–393 (2010).
  Medline CASGoogle Scholar
• 26  Smuga-Otto K, Tian S, Stewart R et al.: Human induced pluripotent stem cells free of vector and transgene sequences. Science324(5928),797–801 (2009).
  MedlineGoogle Scholar
• 27  Kim D, Kim CH, Moon JI et al.: Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cell4(6),472–476 (2009).
  Medline CASGoogle Scholar
• 28  Zhou H, Wu S, Joo JY et al.: Generation of induced pluripotent stem cells using recombinant proteins. Cell Stem Cell4(5),381–384 (2009).
  Medline CASGoogle Scholar
• 29  Li W, Ding S: Small molecules that modulate embryonic stem cell fate and somatic cell reprogramming. Trends Pharm. Sci.31(1),36–45 (2010).
  MedlineGoogle Scholar
• 30  Kim JB, Greber B, Araúzo-Bravo MJ et al.: Direct reprogramming of human neural stem cells by OCT4. Nature461(7264),649–653 (2009).
  Medline CASGoogle Scholar
• 31  Blum B, Benvenisty N: The tumorigenicity of human embryonic stem cells. Adv. Cancer Res.100,133–158 (2008).
  MedlineGoogle Scholar
• 32  Utikal J, Polo JM, Stadtfeld M et al.: Immortalization eliminates a roadblock during cellular reprogramming to iPS cells. Nature460(7259),1145–1148 (2009).▪▪ Faster and more efficient reprogramming of somatic cells to iPS cells is enhanced with knockout of p53, with low endogenous p19 protein levels, pointing to the interplay between reprogramming and cell cycle regulation.
  Medline CASGoogle Scholar
• 33  Hanna J, Saha K, Pando B et al.: Direct cell reprogramming is a stochastic process amenable to acceleration. Nature462(7273),595–601 (2009).
  Medline CASGoogle Scholar
• 34  Okita K, Ichisaka T, Yamanaka S: Generation of germline-competent induced pluripotent stem cells. Nature448(7151),313–317 (2007).
  Medline CASGoogle Scholar
• 35  Wernig M, Meissner A, Foreman R et al.: In vitro reprogramming of fibroblasts into a pluripotent ES-cell-like state. Nature448(7151),318–324 (2007).
  Medline CASGoogle Scholar
• 36  Rossant J: Stem cells: the magic brew. Nature448(7151),260–262 (2007).
  Medline CASGoogle Scholar
• 37  Covin RB, Ambruso DR, England KM et al.: Hypotension and acute pulmonary insufficiency following transfusion of autologous red blood cells during surgery: a case report and review of the literature. Transfus. Med.14(5),375–383 (2004).
  Medline CASGoogle Scholar
• 38  Baussaud V, Mentec H, Fourcade C: Hemolysis after autologous transfusion. Ann. Intern. Med.124(10),931–932 (1996).
  Medline CASGoogle Scholar
• 39  Lleo A, Invernizzi P, Gao B, Podda M, Gershwin ME: Definition of human autoimmunity – autoantibodies versus autoimmune disease. Autoimmun. Rev.9(5),A259–A266 (2010).
  Medline CASGoogle Scholar
• 40  Hanna J, Wernig M, Markoulaki S et al.: Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science318(5858),1920–1923 (2007).▪▪ Use of iPS cell-derived hematopoietic stem cells (and gene therapy) in a humanized mouse model confirms the therapeutic potential of iPS cells for autologous cell therapies to correct genetic diseases, such as sickle cell disease.
  Medline CASGoogle Scholar
• 41  Raya A, Rodríguez-Pizà I, Guenechea G et al.: Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells. Nature460(7251),53–59 (2009).
  Medline CASGoogle Scholar
• 42  Xu D, Alipio Z, Fink LM et al.: Phenotypic correction of murine hemophilia A using an iPS cell-based therapy. Proc. Natl Acad. Sci. USA106(3),808–813 (2009).
  Medline CASGoogle Scholar
• 43  Astermark J, Lacroix-Desmazes S, Reding MT: Inhibitor development. Haemophilia14(Suppl. 3),36–42 (2008).
  Medline CASGoogle Scholar
• 44  Matzner U, Matthes F, Weigelt C et al.: Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease. J. Mol. Med.86(4),433–442 (2008).
  Medline CASGoogle Scholar
• 45  Cideciyan AV, Hauswirth WW, Aleman TS et al.: Vision 1 year after gene therapy for Leber’s congenital amaurosis. N. Engl. J. Med.361(7),725–727 (2009).
  Medline CASGoogle Scholar
• 46  Taylor CJ, Bolton EM, Pocock S, Sharples LD, Pedersen RA, Bradley JA: Banking on human embryonic stem cells: estimating the number of donor cell lines needed for HLA matching. Lancet366(9502),2019–2025 (2005).
  MedlineGoogle Scholar
• 47  Park IH, Arora N, Huo H et al.: Disease-specific induced pluripotent stem cells. Cell134(5),877–886 (2008).
  Medline CASGoogle Scholar
• 48  Dimos JT, Rodolfa KT, Niakan KK et al.: Induced pluripotent stem cells generated from patients with ALS can be differentiated into motor neurons. Science321(5893),1218–1221 (2008).
  Medline CASGoogle Scholar
• 49  Ebert AD, Yu J, Rose FF Jr et al.: Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature457(7227),277–280 (2009).
  Medline CASGoogle Scholar
• 50  Lee G, Papapetrou EP, Kim H et al.: Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs. Nature461(7262),402–406 (2009).▪ Highlights the importance of iPS cell models of disease for elucidating pathophysiology and as a platform for therapeutic drug screens.
  Medline CASGoogle Scholar
• 51  Karumbayaram S, Novitch BG, Patterson M et al.: Directed differentiation of human-induced pluripotent stem cells generates active motor neurons. Stem Cells27(4),806–811 (2009).
  Medline CASGoogle Scholar
• 52  Barber SC, Mead RJ, Shaw PJ: Oxidative stress in ALS: a mechanism of neurodegeneration and a therapeutic target. Biochem. Biophys. Acta1762(11–12),1051–1067 (2006).
  Medline CASGoogle Scholar
• 53  Rothstein JD: Current hypotheses for the underlying biology of amyotropic lateral sclerosis. Ann. Neurol.65(Suppl.),S3–S9 (2009).
  Medline CASGoogle Scholar
• 54  Blurton-Jones M, Kitazawa M, Martinez-Coria H et al.: Neural stem cells improve cognition via BDNF in a transgenic model of Alzheimer disease. Proc. Natl Acad. Sci. USA106(32),13594–13599 (2009).
  Medline CASGoogle Scholar
• 55  Sherer TB, Kim JH, Betarbet R, Greenamyre JT: Subcutaneous rotenone exposure causes highly selective dopaminergic degeneration and α-synuclein aggregation. Exp. Neurol.179(1),9–16 (2003).
  Medline CASGoogle Scholar
• 56  Kaitin KI: Obstacles and opportunities in new drug development. Clin. Pharmacol. Ther.83(2),210–212 (2008).
  Medline CASGoogle Scholar
• 57  So-Tayeb K, Noto FK, Nagaoka M et al.: Highly efficient generation of human hepatocyte-like cells from induced pluripotent stem cells. Hepatology51(1),297–305 (2010).
  MedlineGoogle Scholar
• 58  Sullivan GJ, Hay DC, Park I-H et al.: Generation of functional human hepatic endoderm from human induced pluripotent stem cells. Hepatology51(1),329–335 (2010).
  Medline CASGoogle Scholar
• 59  Song Z, Cai J, Liu Y et al.: Efficient generation of hepatocyte-like cells from human induced pluripotent stem cells. Cell Res.19(11),1233–1242 (2009).
  MedlineGoogle Scholar
• 60  Moriguchi H, Chung RT, Sato C: An identification of the novel combination therapy for hepatitis C virus 1b infection by using a replicon system and human induced pluripotent stem cells. Hepatology51(1),351–352 (2010).
  MedlineGoogle Scholar
• 61  Tanaka T, Tohyama S, Murata M et al.: In vitro pharmacologic testing using human induced pluripotent stem cell-derived cardiomyocytes. Biochem. Biophys. Res. Comm.385(4),497–502 (2009).
  Medline CASGoogle Scholar
• 62  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. Comm.387(3),482–488 (2009).
  Medline CASGoogle Scholar
• 63  Webb S: The gold rush for pluripotent stem cells. Nature27(11),977–979 (2009).
  CASGoogle Scholar
• 64  Ahuja YR, Vijayalakshmi V, Polasa K: Stem cell test: a practical tool in toxicogenomics. Toxicology231(1),1–10 (2007).
  Medline CASGoogle Scholar
• 65  Zarzeczny A, Scott C, Hyun I et al.: iPS cells: mapping the policy issues. Cell139(6),1032–1037 (2009).
  Medline CASGoogle Scholar
• 66  Condic ML, Rao M: Regulatory issues for personalized pluripotent cells. Stem Cells26(11),2753–2758 (2008).
  Medline CASGoogle Scholar
• 67  Vrtovec KT, Scott CT: Patenting pluripotence: the next battle for stem cell intellectual property. Nat. Biotechnol.26(4),393–395 (2008).▪ A review of conflicting claims on iPS cell technology that may impact translation of iPS cell-based therapies.
  Medline CASGoogle Scholar
• 68  Halme DG, Kessler DA: FDA regulation of stem-cell-based therapies. N. Engl. J. Med.355(16),1730–1735 (2006).
  Medline CASGoogle Scholar
• 69  Taylor DA: From stem cells and cadaveric matrix to engineered organs. Curr. Opin. Biotechnol.20(5),598–605 (2009).
  Medline CASGoogle Scholar
• 70  Yamanaka S: A fresh look at iPS cells. Cell137(1),13–17 (2009).
  Medline CASGoogle Scholar
• 71  Kaufman DS: Toward clinical therapies using hematopoietic cells derived from human pluripotent stem cells. Blood114(17),3513–3523 (2009).
  Medline CASGoogle Scholar
• 72  Hirami Y, Osakada F, Takahashi K et al.: Generation of retinal cells from mouse and human induced pluripotent stem cells. Neurosci. Lett.458(3),126–131 (2009).
  Medline CASGoogle Scholar
• 73  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 One4(12),e8152 (2009).
  MedlineGoogle Scholar
• 74  Buchholz DE, Hikita ST, Rowland TJ et al.: Derivation of functional retinal pigmented epithelium from induced pluripotent stem cells. Stem Cells27(10),2427–2434 (2009).
  Medline CASGoogle Scholar
• 75  Tezel TH, Del Priore LV, Berger AS, Kaplan HJ: Adult retinal pigment epithelial transplantation in exudative age-related macular degeneration. Am. J. Ophthal.143(4),584–595 (2007).
  MedlineGoogle Scholar
• 76  Ameri H, Ratanapakorn T, Ufer S et al.: Toward a wide-field retinal prosthesis. J. Neural Eng.6(3),035002 (2009).
  MedlineGoogle Scholar
• 77  Coghlan A: Stem cell eye ‘patch’ to save sight gets a cash boost. New Scientist 24th April (2009).
  Google Scholar
• 78  Lu B, Malcuit C, Wang S et al.: Long-term safety and function of RPE from human embryonic stem cells in preclinical models of macular degeneration. Stem Cells27(9),2126–2135.
  MedlineGoogle Scholar
• 101  Geron: human embryonic stem cells – derived therapies www.geron.com/patients/clinicaltrials/hESC.aspx
  Google Scholar
• 102  Advanced Cell Technology www.advancedcell.com
  Google Scholar