Ophthalmologic stem cell transplantation therapies

Regenerative Medicine

Volumn 7, Issue 6 SUPPL., 2012, Pages 32-39

  Blenkinsop, Timothy A ^a   Corneo, Barbara ^a   Temple, Sally ^a   Stern, Jeffrey H ^a  

^a Regenerative Research Foundation   (United States)

Author keywords

Bone marrow stem cell; Eye disease; Human embryonic stem cell; Human neural stem cell; Induced pluripotent stem cell; Limbal stem cell; Retinal pigment epithelial stem cell; Umbilical cord stem cell

Indexed keywords

ARTICLE; BONE MARROW TRANSPLANTATION; CLINICAL EFFECTIVENESS; CORD BLOOD STEM CELL TRANSPLANTATION; CORNEA TRANSPLANTATION; EYE DISEASE; HUMAN; LIMBAL STEM CELL TRANSPLANTATION; NEURAL STEM CELL TRANSPLANTATION; PATIENT SAFETY; PHOTORECEPTOR CELL; PIGMENT EPITHELIUM; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; RETINA GYRATE ATROPHY; RETINA MACULA AGE RELATED DEGENERATION; RETINA PIGMENT DEGENERATION; RETINITIS PIGMENTOSA; STEM CELL TRANSPLANTATION; TISSUE ENGINEERING;

ANIMALS; CLINICAL TRIALS AS TOPIC; CORNEA; EYE DISEASES; HUMANS; PHOTORECEPTOR CELLS, VERTEBRATE; RETINAL PIGMENT EPITHELIUM; STEM CELL TRANSPLANTATION;

EID: 84870694552     PISSN: 17460751     EISSN: 1746076X     Source Type: Journal    
DOI: 10.2217/rme.12.77     Document Type: Article

References (65)

1. Braunstein R. AMD: its potential health and economic impact around the world. World Ophthalmology Congress Abstracts, ISRET- SA 232 (2012).
2. Chaudhry GR, Fecek C, Lai MM et al. Fate of embryonic stem cell derivatives implanted into the vitreous of a slow retinal degenerative mouse model. Stem Cells Dev. 18(2), 247-258 (2009).
3. Li Y, Zhong X, Yan J et al. Pluripotent embryonic stem cells developed into medulloepithelioma in nude mice eyes. Yan Ke Xue Bao 18(1), 37-44 (2002).
4. Arnhold S, Klein H, Semkova I, Addicks K, Schraermeyer U. Neurally selected embryonic stem cells induce tumor formation after long-term survival following engraftment into the subretinal space. Invest. Ophthalmol. Vis. Sci. 45(12), 4251-4255 (2004).
5. Van Der Bogt KE, Swijnenburg Rj, Cao F, Wu JC. Molecular imaging of human embryonic stem cells: keeping an eye on differentiation, tumorigenicity and immunogenicity. Cell Cycle 5(23), 2748-2752 (2006).
6. US FDA Cellular, Tissue and Gene Therapies Advisory Committee. Cellular and Gene Therapies for Retinal Disorders (CTGTAC Meeting #52). Cellular, Tissue and Gene Therapies Advisory Committee, FDA, USA (2011).
7. Robinton DA, Daley GQ. The promise of induced pluripotent stem cells in research and therapy. Nature 481(7381), 295-305 (2012).
8. Takahashi K, Tanabe K, Ohnuki M et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131(5), 861-872 (2007).
9. Takahashi K, Yamanaka S. Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors. Cell 126(4), 663-676 (2006).
10. Mostoslavsky G. Concise review: the magic act of generating induced pluripotent stem cells: many rabbits in the hat. Stem Cells 30(1), 28-32 (2012).
11. Aboody K, Capela A, Niazi N, Stern JH, Temple S. Translating stem cell studies to the clinic for CNS repair: current state of the art and the need for a Rosetta Stone. Neuron 70(4), 597-613 (2011).
12. Salero E, Blenkinsop TA, Corneo B et al. Adult human RPE can be activated into a multipotent stem cell that produces mesenchymal derivatives. Cell Stem Cell 10(1), 88-95 (2012).
13. O'Callaghan AR, Daniels JT. Concise review: limbal epithelial stem cell therapy: controversies and challenges. Stem Cells 29(12), 1923-1932 (2011).
14. Friedlander M, Dorrell MI, Ritter MR et al. Progenitor cells and retinal angiogenesis. Angiogenesis 10(2), 89-101 (2007).
15. Lim LS, Mitchell P, Seddon JM, Holz FG, Wong TY. Age-related macular degeneration. Lancet 379(9827), 1728-1738 (2012).
16. Strauss O. The retinal pigment epithelium in visual function. Physiol. Rev. 85(3), 845-881 (2005).
17. Bonilha Vl, Rayborn ME, Bhattacharya SK et al. The retinal pigment epithelium apical microvilli and retinal function. Adv. Exp. Med. Biol. 572, 519-524 (2006).
18. Sparrow JR, Hicks D, Hamel CP. The retinal pigment epithelium in health and disease. Curr. Mol. Med. 10(9), 802-823 (2010).
19. Bharti K, Miller SS, Arnheiter H. The new paradigm: retinal pigment epithelium cells generated from embryonic or induced pluripotent stem cells. Pigment Cell Melanoma Res. 24(1), 21-34 (2011).
20. Klein R, Chou CF, Klein BE, Zhang X, Meuer SM, Saaddine JB. Prevalence of agerelated macular degeneration in the US population. Arch. Ophthalmol. 129(1), 75-80 (2011).
21. Del Priore LV, Tezel TH, Kaplan HJ. Maculoplasty for age-related macular degeneration: reengineering Bruch's membrane and the human macula. Prog. Retin. Eye Res. 25(6), 539-562 (2006).
22. Da Cruz L, Chen FK, Ahmado A, Greenwood J, Coffey P. RPE transplantation and its role in retinal disease. Prog. Retin. Eye Res. 26(6), 598-635 (2007).
23. Binder S, Stanzel BV, Krebs I, Glittenberg C. Transplantation of the RPE in AMD. Prog. Retin. Eye Res. 26(5), 516-554 (2007).
24. Tezel TH, Del Priore LV, Berger AS, Kaplan HJ. Adult retinal pigment epithelial transplantation in exudative age-related macular degeneration. Am. J. Ophthalmol. 143(4), 584-595 (2007).
25. Klimanskaya I, Hipp J, Rezai KA, West M, Atala A, Lanza R. Derivation and comparative assessment of retinal pigment epithelium from human embryonic stem cells using transcriptomics. Cloning Stem Cells 6(3), 217-245 (2004).
26. Lund Rd, Wang S, Klimanskaya I et al. Human embryonic stem cell-derived cells rescue visual function in dystrophic RCS rats. Cloning Stem Cells 8(3), 189-199 (2006).
27. Corneo B, Temple S. Sense and serendipity aid RPE generation. Cell Stem Cell 5(4), 347-348 (2009).
28. Osakada F, Ikeda H, Mandai M et al. Toward the generation of rod and cone photoreceptors from mouse, monkey and human embryonic stem cells. Nat. Biotechnol. 26(2), 215-224 (2008).
29. 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).
30. Idelson M, Alper R, Obolensky A et al. Directed differentiation of human embryonic stem cells into functional retinal pigment epithelium cells. Cell Stem Cell 5(4), 396-408 (2009).
31. Osakada F, Jin Zb, Hirami Y et al. In vitro differentiation of retinal cells from human pluripotent stem cells by smallmolecule induction. J. Cell. Sci. 122(Pt 17), 3169-3179 (2009).
32. Rowland TJ, Buchholz DE, Clegg DO. Pluripotent human stem cells for the treatment of retinal disease. J. Cell Physiol. 227(2), 457-466 (2012).
33. 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 Cells 27(9), 2126-2135 (2009).
34. Schwartz SD, Hubschman JP, Heilwell G et al. Embryonic stem cell trials for macular degeneration: a preliminary report. Lancet 379(9817), 713-720 (2012).
35. Taylor AW, Kaplan HJ. Ocular immune privilege in the year 2010: ocular immune privilege and uveitis. Ocul. Immunol. Inflamm. 18(6), 488-492 (2010).
36. Carr Aj, Vugler A, Lawrence J et al. Molecular characterization and functional analysis of phagocytosis by human embryonic stem cell-derived RPE cells using a novel human retinal assay. Mol. Vis. 15, 283-295 (2009).
37. 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. 214(2), 347-361 (2008).
38. Jin Zb, Okamoto S, Osakada F et al. Modeling retinal degeneration using patient-specific induced pluripotent stem cells. PLoS ONE 6(2), e17084 (2011).
39. Mcgill TJ, Cottam B, Lu B et al. Transplantation of human central nervous system stem cells - neuroprotection in retinal degeneration. Eur. J. Neurosci. 35(3), 468-477 (2012).
40. Lund RD, Wang S, Lu B et al. Cells isolated from umbilical cord tissue rescue photoreceptors and visual functions in a rodent model of retinal disease. Stem Cells 25(3), 602-611 (2007).
41. Arnhold S, Absenger Y, Klein H, Addicks K, Schraermeyer U. Transplantation of bone marrow-derived mesenchymal stem cells rescue photoreceptor cells in the dystrophic retina of the rhodopsin knockout mouse. Graefes Arch. Clin. Exp. Ophthalmol. 245(3), 414-422 (2007).
42. Otani A, Dorrell Mi, Kinder K et al. Rescue of retinal degeneration by intravitreally injected adult bone marrow-derived lineagenegative hematopoietic stem cells. J. Clin. Invest. 114(6), 765-774 (2004).
43. Siqueira RC, Messias A, Voltarelli JC, Scott IU, Jorge R. Intravitreal injection of autologous bone marrow-derived mononuclear cells for hereditary retinal dystrophy: a Phase I trial. Retina 31(6), 1207-1214 (2011).
44. Moore AT. Cone and cone-rod dystrophies. J. Med. Genet. 29(5), 289-290 (1992).
45. Pellegrini G, Traverso CE, Franzi AT, Zingirian M, Cancedda R, De Luca M. Long-term restoration of damaged corneal surfaces with autologous cultivated corneal epithelium. Lancet 349(9057), 990-993 (1997).
46. Rama P, Bonini S, Lambiase A et al. Autologous fibrin-cultured limbal stem cells permanently restore the corneal surface of patients with total limbal stem cell deficiency. Transplantation 72(9), 1478-1485 (2001).
47. Rama P, Matuska S, Paganoni G, Spinelli A, De Luca M, Pellegrini G. Limbal stemcell therapy and long-term corneal regeneration. N. Engl. J. Med. 363(2), 147-155 (2010).
48. Wylegala E, Dobrowolski D, Tarnawska D et al. Limbal stem cells transplantation in the reconstruction of the ocular surface: 6 years experience. Eur. J. Ophthalmol. 18(6), 886-890 (2008).
49. Mohand-Said S, Hicks D, Simonutti M et al. Photoreceptor transplants increase host cone survival in the retinal degeneration (rd) mouse. Ophthalmic Res. 29(5), 290-297 (1997).
50. Kwan AS, Wang S, Lund RD. Photoreceptor layer reconstruction in a rodent model of retinal degeneration. Exp. Neurol. 159(1), 21-33 (1999).
51. Lamba DA, Gust J, Reh TA. Transplantation of human embryonic stem cell-derived photoreceptors restores some visual function in Crx-deficient mice. Cell Stem Cell 4(1), 73-79 (2009).
52. West EL, Pearson RA, Barker SE et al. Long-term survival of photoreceptors transplanted into the adult murine neural retina requires immune modulation. Stem Cells 28(11), 1997-2007 (2010).
53. Phillips JB, Blanco-Sanchez B, Lentz JJ et al. Harmonin (Ush1c) is required in zebrafish Muller glial cells for photoreceptor synaptic development and function. Dis. Model Mech. 4(6), 786-800 (2012).
54. Lamba DA, Reh TA. Microarray characterization of human embryonic stem cell - derived retinal cultures. Invest. Ophthalmol. Vis. Sci. 52(7), 4897-4906 (2011).
55. Yue F, Johkura K, Shirasawa S et al. Differentiation of primate ES cells into retinal cells induced by ES cell-derived pigmented cells. Biochem. Biophys. Res. Commun. 394(4), 877-883 (2010).
56. Eiraku M, Takata N, Ishibashi H et al. Self-organizing optic-cup morphogenesis in three-dimensional culture. Nature 472(7341), 51-56 (2011).
57. Nakano T, Ando S, Takata N et al. Selfformation of optic cups and storable stratified neural retina from human ESCs. Cell Stem Cell 10(6), 771-785 (2012).
58. Hynes SR, Lavik EB. A tissue-engineered approach towards retinal repair: scaffolds for cell transplantation to the subretinal space. Graefes Arch. Clin. Exp. Ophthalmol. 248(6), 763-778 (2010).
59. Castellarin AA, Nasir M, Sugino IK, Zarbin MA. Progressive presumed choriocapillaris atrophy after surgery for age-related macular degeneration. Retina 18(2), 143-149 (1998).
60. Sodha S, Wall K, Redenti S, Klassen H, Young MJ, Tao SL. Microfabrication of a three-dimensional polycaprolactone thinfilm scaffold for retinal progenitor cell encapsulation. J. Biomater. Sci. Polym. Ed. 22(4-6), 443-456 (2012).
61. Mcusic AC, Lamba DA, Reh TA. Guiding the morphogenesis of dissociated newborn mouse retinal cells and hES cell-derived retinal cells by soft lithography-patterned microchannel PLGA scaffolds. Biomaterials 33(5), 1396-1405 (2011).
62. Thieltges F, Stanzel BV, Liu Z, Holz FG. A nanofibrillar surface promotes superior growth characteristics in cultured human retinal pigment epithelium. Ophthalmic Res. 46(3), 133-140 (2011).
63. Simell O, Takki K. Raised plasmaornithine and gyrate atrophy of the choroid and retina. Lancet 1(7811), 1031-1033 (1973).
64. Meyer JS, Howden SE, Wallace KA et al. Optic vesicle-like structures derived from human pluripotent stem cells facilitate a customized approach to retinal disease treatment. Stem Cells 29(8), 1206-1218 (2011).
65. 101 Advanced Cell Technology www.advancedcell.com