Generation of retinal pigment epithelial cells from small molecules and OCT4 reprogrammed human induced pluripotent stem cells

Stem Cells Translational Medicine

Volumn 1, Issue 2, 2012, Pages 96-109

  Krohne, Tim U ^a   Westenskow, Peter D ^a   Kurihara, Toshihide ^a   Friedlander, David F ^a   Lehmann, Mandy ^a   Dorsey, Alison L ^a   Li, Wenlin ^a   Zhu, Saiyong ^a   Schultz, Andrew ^a   Wang, Junhua ^a   Siuzdak, Gary ^a   Ding, Sheng ^a   Friedlander, Martin ^a  

^a SCRIPPS RESEARCH INSTITUTE   (United States)

Author keywords

Aging; Differentiation; Induced pluripotent stem cells; Retina; Small molecules; Stem cell transplantation

Indexed keywords

4 [4 (1, 3 BENZODIOXOL 5 YL) 5 (2 PYRIDINYL) 1H IMIDAZOL 2 YL] BENZAMIDE; KRUPPEL LIKE FACTOR 4; LIPOFUSCIN; MICROPHTHALMIA ASSOCIATED TRANSCRIPTION FACTOR; MONOPHENOL MONOOXYGENASE; OCTAMER TRANSCRIPTION FACTOR 4; PROTEIN ZO1; RETINOID BINDING PROTEIN; TRANSCRIPTION FACTOR NANOG; TRANSCRIPTION FACTOR OTX2; TRANSCRIPTION FACTOR SOX2; TRANYLCYPROMINE; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL DIFFERENTIATION; CONFOCAL MICROSCOPY; CONTROLLED STUDY; ELECTRON MICROSCOPY; ELECTRORETINOGRAPHY; ENZYME LINKED IMMUNOSORBENT ASSAY; FIBROBLAST; FLUORESCENCE MICROSCOPY; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; HISTOLOGY; HUMAN; HUMAN CELL; IMMUNOCYTOCHEMISTRY; IMMUNOHISTOCHEMISTRY; KERATINOCYTE; MASS SPECTROMETRY; NONHUMAN; OPTICAL COHERENCE TOMOGRAPHY; PHAGOCYTOSIS; PHOTORECEPTOR OUTER SEGMENT; PIGMENT EPITHELIUM; PLURIPOTENT STEM CELL; PROTEIN EXPRESSION; RAT; RETINA DEGENERATION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION;

EID: 84860622908     PISSN: 21576564     EISSN: 21576580     Source Type: Journal    
DOI: 10.5966/sctm.2011-0057     Document Type: Article

References (57)

1. Yu J, Vodyanik MA, Smuga-Otto K et al. Induced pluripotent stem cell lines derived from human somatic cells. Science 2007;318: 1917-1920.
2. 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.
3. Koch P, Kokaia Z, Lindvall O et al. Emerging concepts in neural stem cell research: Autologous repair and cell-based disease modelling. Lancet Neurol 2009;8:819-829.
4. Aoi T, Yae K, Nakagawa M et al. Generation of pluripotent stem cells from adult mouse liver and stomach cells. Science 2008;321: 699-702.
5. Okita K, Ichisaka T, Yamanaka S. Generation of germline-competent induced pluripotent stem cells. Nature 2007;448:313-317.
6. Kim D, Kim CH, Moon JI et al. Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins. Cell Stem Cell 2009;4:472-476.
7. Markoulaki S, Hanna J, Beard C et al. Transgenic mice with defined combinations of drug-inducible reprogramming factors. Nat Biotechnol 2009;27:169-171.
8. Miura K, Okada Y, Aoi T et al. Variation in the safety of induced pluripotent stem cell lines. Nat Biotechnol 2009;27:743-745.
9. Bock C, Kiskinis E, Verstappen G et al. Reference Maps of human ES and iPS cell variation enable high-throughput characterization of pluripotent cell lines. Cell 2011;144:439-452.
10. Hussein SM, Batada NN, Vuoristo S et al. Copy number variation and selection during reprogramming to pluripotency. Nature 2011; 471:58-62.
11. Laurent LC, Ulitsky I, Slavin I et al. Dynamic changes in the copy number of pluripotency and cell proliferation genes in human ESCs and iPSCs during reprogramming and time in culture. Cell Stem Cell 2011;8:106-118.
12. Mayshar Y, Ben-David U, Lavon N et al. Identification and classification of chromosomal aberrations in human induced pluripotent stem cells. Cell Stem Cell 2010;7:521-531.
13. Li W, Zhou H, Abujarour R et al. Generation of human-induced pluripotent stem cells in the absence of exogenous Sox2. STEM CELLS 2009;27:2992-3000.
14. Zhu S, Li W, Zhou H et al. Reprogramming of human primary keratinocytes by Oct4 with chemical compounds. Cell Stem Cell 2010;7: 651-655.
15. Strauss O. The retinal pigment epithelium in visual function. Physiol Rev 2005;85: 845-881.
16. Congdon N, O'Colmain B, Klaver CC et al. Causes and prevalence of visual impairment among adults in the United States. Arch Ophthalmol 2004;122:477-485.
17. Resnikoff S, Pascolini D, Etya'ale D et al. Global data on visual impairment in the year 2002. Bull World Health Organ 2004;82:844-851.
18. Cahill MT, Freedman SF, Toth CA. Macular translocation with 360 degrees peripheral retinectomy for geographic atrophy. Arch Ophthalmol 2003;121:132-133.
19. Joussen AM, Joeres S, Fawzy N et al. Autologous translocation of the choroid and retinal pigment epithelium in patients with geographic atrophy. Ophthalmology 2007;114: 551-560.
20. Idelson M, Alper R, Obolensky A et al. Directed differentiation of human embryonic stem cells into functional retinal pigment epithelium cells. Cell Stem Cell 2009;5:396-408.
21. Vugler A, Carr AJ, Lawrence J et al. Elucidating the phenomenon of HESC-derived RPE: Anatomy of cell genesis, expansion and retinal transplantation. Exp Neurol 2008;214:347-361.
22. Buchholz DE, Hikita ST, Rowland TJ et al. Derivation of functional retinal pigmented epithelium from induced pluripotent stem cells. STEM CELLS 2009;27:2427-2434.
23. 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;4:e8152.
24. Meyer JS, Shearer RL, Capowski EE et al. Modeling early retinal development with human embryonic and induced pluripotent stem cells. Proc Natl Acad Sci U S A 2009;106: 16698-16703.
25. Osakada F, Jin ZB, Hirami Y et al. In vitro differentiation of retinal cells from human pluripotent stem cells by small-molecule induction. J Cell Sci 2009;122:3169-3179.
26. Hirami Y, Osakada F, Takahashi K et al. Generation of retinal cells from mouse and human induced pluripotent stem cells. Neurosci Lett 2009;458:126-131.
27. Liao JL, Yu J, Huang K et al. Molecular signature of primary retinal pigment epithelium and stem-cell-derived RPE cells. Hum Mol Genet 2010;19:4229-4238.
28. 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.
29. Cai J, Chen J, Liu Y et al. Assessing selfrenewal and differentiation in human embryonic stem cell lines. STEM CELLS 2006;24:516-530.
30. Krohne TU, Holz FG, Kopitz J. Apical-tobasolateral transcytosis of photoreceptor outer segments induced by lipid peroxidation products in human retinal pigment epithelial cells. Invest Ophthalmol Vis Sci 2010;51:553-560.
31. Parish CA, Hashimoto M, Nakanishi K et al. Isolation and one-step preparation of A2E and iso-A2E, fluorophores from human retinal pigment epithelium. Proc Natl Acad Sci U S A 1998;95:14609-14613.
32. Wu YL, Yanase E, Feng XD et al. Structural characterization of bisretinoid A2E photocleavage products and implications for age-related macular degeneration. Proc Natl Acad Sci USA2010;107:7275-7280.
33. Smith CA, Want EJ, O'Maille G et al. XCMS: Processing mass spectrometry data for metabolite profiling using nonlinear peak alignment, matching, and identification. Anal Chem 2006;78:779-787.
34. O'Rahilly R. The prenatal development of the human eye. Exp Eye Res 1975;21:93-112.
35. Mircheff AK, Miller SS, Farber DB et al. Isolation and provisional identification of plasma membrane populations from cultured human retinal pigment epithelium. Invest Ophthalmol Vis Sci 1990;31:863-878.
36. Young RW, Bok D. Participation of the retinal pigment epithelium in the rod outer segment renewal process. J Cell Biol 1969;42: 392-403.
37. Ohno-Matsui K, Morita I, Tombran-Tink J et al. Novel mechanism for age-related macular degeneration: An equilibrium shift between the angiogenesis factors VEGF and PEDF. J Cell Physiol 2001;189:323-333.
38. Sonoda S, Sreekumar PG, Kase S et al. Attainment of polarity promotes growth factor secretion by retinal pigment epithelial cells: Relevance to age-related macular degeneration. Aging (Albany NY) 2010;2:28-42.
39. Zhu D, Deng X, Spee C et al. Polarized secretion of PEDF from human embryonic stem cell-derived RPE promotes retinal progenitor cell survival. Invest Ophthalmol Vis Sci 2011;52:1573-1585.
40. Holz FG, Bellman C, Staudt S et al. Fundus autofluorescence and development of geographic atrophy in age-related macular degeneration. Invest Ophthalmol Vis Sci 2001;42: 1051-1056.
41. von Rückmann A, Fitzke FW, Bird AC. In vivo fundus autofluorescence in macular dystrophies. Arch Ophthalmol 1997;115:609-615.
42. Sparrow JR, Yoon KD, Wu Y et al. Interpretations of fundus autofluorescence from studies of the bisretinoids of the retina. Invest Ophthalmol Vis Sci 2010;51:4351-4357.
43. Charbel Issa P, Bolz HJ, Ebermann I et al. Characterisation of severe rod-cone dystrophy in a consanguineous family with a splice site mutation in the MERTK gene. Br J Ophthalmol 2009;93:920-925.
44. Delori FC, Dorey CK, Staurenghi G et al. In vivo fluorescence of the ocular fundus exhibits retinal pigment epithelium lipofuscin characteristics. Invest Ophthalmol Vis Sci 1995;36: 718-729.
45. Eldred GE, Lasky MR. Retinal age pigments generated by self-assembling lysosomotropic detergents. Nature 1993;361:724-726.
46. Liu J, Itagaki Y, Ben-Shabat S et al. The biosynthesis of A2E, a fluorophore of aging retina, involves the formation of the precursor, A2-PE, in the photoreceptor outer segment membrane. J Biol Chem 2000;275:29354-29360.
47. Gupta N, Brown KE, Milam AH. Activated microglia in human retinitis pigmentosa, lateonset retinal degeneration, and age-related macular degeneration. Exp Eye Res 2003;76: 463-471.
48. Harada T, Harada C, Kohsaka S et al. Microglia-Muller glia cell interactions control neurotrophic factor production during light-induced retinal degeneration. J Neurosci 2002; 22:9228-9236.
49. Lewis GP, Sethi CS, Carter KM et al. Microglial cell activation following retinal detachment: A comparison between species. Mol Vis 2005;11:491-500.
50. Katz ML, Redmond TM. Effect of Rpe65 knockout on accumulation of lipofuscin fluorophores in the retinal pigment epithelium. Invest Ophthalmol Vis Sci 2001;42:3023-3030.
51. Kim SR, Fishkin N, Kong J et al. Rpe65 Leu450Met variant is associated with reduced levels of the retinal pigment epithelium lipofuscin fluorophores A2E and iso-A2E. Proc Natl Acad Sci U S A 2004;101:11668-11672.
52. Thomas BB, Arai S, Ikai Y et al. Retinal transplants evaluated by optical coherence tomography in photoreceptor degenerate rats. J Neurosci Methods 2006;151:186-193.
53. Seddon JM, Reynolds R, Maller J et al. Prediction model for prevalence and incidence of advanced age-related macular degeneration based on genetic, demographic, and environmental variables. Invest Ophthalmol Vis Sci 2009;50:2044-2053.
54. Baker M. Metabolomics: From small molecules to big ideas. Nat Methods 2011;8: 117-121.
55. Wikoff WR, Kalisak E, Trauger S et al. Response and recovery in the plasma metabolome tracks the acute LCMV-induced immune response. J Proteome Res 2009;8: 3578-3587.
56. Yanes O, Clark J, Wong DM et al. Metabolic oxidation regulates embryonic stem cell differentiation. Nat Chem Biol 2010;6:411-417.
57. Marc RE, Jones BW, Watt CB et al. Extreme retinal remodeling triggered by light damage: Implications for age related macular degeneration. Mol Vis 2008;14:782-806.