Using human induced pluripotent stem cells to treat retinal disease

Progress in Retinal and Eye Research - Tập 37 - Trang 163-181 - 2013
Shyamanga Borooah1,2, M. Joseph Phillips3, Bilada Bilican1, Alan F. Wright4, B. Willke1, Siddharthan Chandran1, David M. Gamm5,3, Baljean Dhillon2
1MRC Centre for Regenerative Medicine, University of Edinburgh, Edinburgh EH16 4UU, UK
2Ophthalmology, School of Clinical Sciences, University of Edinburgh, Edinburgh EH16 4SB, UK
3Waisman Center, University of Wisconsin School of Medicine and Public Health, 1500 Highland Ave, Madison, WI 53705, USA
4MRC Human Genetics Unit, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh EH4 2XU, UK
5Department of Ophthalmology and Visual Sciences, McPherson Eye Research Institute, University of Wisconsin School of Medicine and Public Health, 1500 Highland Ave, Madison, WI 53705, USA

Tóm tắt

Từ khóa


Tài liệu tham khảo

Aasen, 2008, Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes, Nat. Biotechnol., 26, 1276, 10.1038/nbt.1503

Bae, 2012, Hypoxia enhances the generation of retinal progenitor cells from human induced pluripotent and embryonic stem cells, Stem Cells Dev., 21, 1344, 10.1089/scd.2011.0225

Balse, 2005, Purification of mammalian cone photoreceptors by lectin panning and the enhancement of their survival in glia-conditioned medium, Invest. Ophthalmol. Vis. Sci., 46, 367, 10.1167/iovs.04-0695

Bar-Nur, 2011, Epigenetic memory and preferential lineage-specific differentiation in induced pluripotent stem cells derived from human pancreatic islet beta cells, Cell Stem Cell, 9, 17, 10.1016/j.stem.2011.06.007

Barber, 2013, Repair of the degenerate retina by photoreceptor transplantation, Proc. Natl. Acad. Sci. U. S. A., 110, 354, 10.1073/pnas.1212677110

Ben-David, 2011, Large-scale analysis reveals acquisition of lineage-specific chromosomal aberrations in human adult stem cells, Cell Stem Cell, 9, 97, 10.1016/j.stem.2011.06.013

Bharti, 2011, The new paradigm: retinal pigment epithelium cells generated from embryonic or induced pluripotent stem cells, Pigment Cell Melanoma Res., 24, 21, 10.1111/j.1755-148X.2010.00772.x

Bilican, 2013, Comment on “Drug screening for ALS using patient-specific induced pluripotent stem cells”, Sci. Transl. Med., 5, 188l, 10.1126/scitranslmed.3005065

Bock, 2011, Reference Maps of human ES and iPS cell variation enable high-throughput characterization of pluripotent cell lines, Cell, 144, 439, 10.1016/j.cell.2010.12.032

Brambrink, 2008, Sequential expression of pluripotency markers during direct reprogramming of mouse somatic cells, Cell Stem Cell, 2, 151, 10.1016/j.stem.2008.01.004

Buchholz, 2009, Derivation of functional retinal pigmented epithelium from induced pluripotent stem cells, Stem Cells, 27, 2427, 10.1002/stem.189

Buchholz, 2013, Rapid and efficient directed differentiation of human pluripotent stem cells into retinal pigmented epithelium, Stem Cells Transl. Med., 2, 384, 10.5966/sctm.2012-0163

Byrne, 2009, Enhanced generation of induced pluripotent stem cells from a subpopulation of human fibroblasts, PLoS One, 4, e7118, 10.1371/journal.pone.0007118

Carr, 2009, Protective effects of human iPS-derived retinal pigment epithelium cell transplantation in the retinal dystrophic rat, PLoS One, 4, e8152, 10.1371/journal.pone.0008152

Cermak, 2011, Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting, Nucleic Acids Res., 39, e82, 10.1093/nar/gkr218

Chambers, 2009, Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling, Nat. Biotechnol., 27, 275, 10.1038/nbt.1529

Chan, 2009, Live cell imaging distinguishes bona fide human iPS cells from partially reprogrammed cells, Nat. Biotechnol., 27, 1033, 10.1038/nbt.1580

Chen, 2011, Chemically defined conditions for human iPSC derivation and culture, Nat. Methods, 8, 424, 10.1038/nmeth.1593

Chen, 2012, Serum starvation induced cell cycle synchronization facilitates human somatic cells reprogramming, PLoS One, 7, e28203, 10.1371/journal.pone.0028203

Cheong, 1993, Birth of mice after transplantation of early cell-cycle-stage embryonic nuclei into enucleated oocytes, Biol. Reprod., 48, 958, 10.1095/biolreprod48.5.958

Cimadamore, 2009, Nicotinamide rescues human embryonic stem cell-derived neuroectoderm from parthanatic cell death, Stem Cells, 27, 1772, 10.1002/stem.107

Cyranoski, 2013, Stem cells cruise to clinic, Nature, 494, 413, 10.1038/494413a

Davis, 1987, Expression of a single transfected cDNA converts fibroblasts to myoblasts, Cell, 51, 987, 10.1016/0092-8674(87)90585-X

Davis, 1989, MyoD and achaete-scute: 4-5 amino acids distinguishes myogenesis from neurogenesis, Princess Takamatsu Symp., 20, 267

Di Giorgio, 2007, Non-cell autonomous effect of glia on motor neurons in an embryonic stem cell-based ALS model, Nat. Neurosci., 10, 608, 10.1038/nn1885

Diniz, 2013, Subretinal implantation of retinal pigment epithelial cells derived from human embryonic stem cells – improved survival when implanted as a monolayer, Invest. Ophthalmol. Vis. Sci., 54, 5087, 10.1167/iovs.12-11239

Eberle, 2011, Increased integration of transplanted CD73-positive photoreceptor precursors into adult mouse retina, Invest. Ophthalmol. Vis. Sci., 52, 6462, 10.1167/iovs.11-7399

Eiraku, 2011, Self-organizing optic-cup morphogenesis in three-dimensional culture, Nature, 472, 51, 10.1038/nature09941

Eminli, 2009, Differentiation stage determines potential of hematopoietic cells for reprogramming into induced pluripotent stem cells, Nat. Genet., 41, 968, 10.1038/ng.428

Erecinska, 2001, Tissue oxygen tension and brain sensitivity to hypoxia, Respir. Physiol., 128, 263, 10.1016/S0034-5687(01)00306-1

Esteban, 2010, Vitamin C enhances the generation of mouse and human induced pluripotent stem cells, Cell Stem Cell, 6, 71, 10.1016/j.stem.2009.12.001

Fusaki, 2009, Efficient induction of transgene-free human pluripotent stem cells using a vector based on Sendai virus, an RNA virus that does not integrate into the host genome, Proc. Jpn. Acad. Ser. B Phys. Biol. Sci., 85, 348, 10.2183/pjab.85.348

Gamm, 2008, A novel serum-free method for culturing human prenatal retinal pigment epithelial cells, Invest. Ophthalmol. Vis. Sci., 49, 788, 10.1167/iovs.07-0777

Garita-Hernandez, 2013, Hypoxia increases the yield of photoreceptors differentiating from mouse embryonic stem cells and improves the modeling of retinogenesis in vitro, Stem Cells, 10.1002/stem.1339

Geti, 2012, A practical and efficient cellular substrate for the generation of induced pluripotent stem cells from adults: blood-derived endothelial progenitor cells, Stem Cells Transl. Med., 1, 855, 10.5966/sctm.2012-0093

Giorgetti, 2009, Generation of induced pluripotent stem cells from human cord blood using OCT4 and SOX2, Cell Stem Cell, 5, 353, 10.1016/j.stem.2009.09.008

Gonzalez-Cordero, 2013, Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina, Nat. Biotechnol., 31, 741, 10.1038/nbt.2643

Gotz, 2005, The cell biology of neurogenesis, Nat. Rev. Mol. Cell. Biol., 6, 777, 10.1038/nrm1739

Gourraud, 2012, The role of human leukocyte antigen matching in the development of multiethnic “haplobank” of induced pluripotent stem cell lines, Stem Cells, 30, 180, 10.1002/stem.772

Gundry, 2012, A cell surfaceome map for immunophenotyping and sorting pluripotent stem cells, Mol. Cell. Proteomics, 11, 303, 10.1074/mcp.M112.018135

Gurdon, 1962, The developmental capacity of nuclei taken from intestinal epithelium cells of feeding tadpoles, J. Embryol. Exp. Morphol., 10, 622

Gust, 2011, Adult donor rod photoreceptors integrate into the mature mouse retina, Invest. Ophthalmol. Vis. Sci., 52, 5266, 10.1167/iovs.10-6329

Hambright, 2012, Long-term survival and differentiation of retinal neurons derived from human embryonic stem cell lines in un-immunosuppressed mouse retina, Mol. Vis., 18, 920

Han, 2012, Direct reprogramming of fibroblasts into neural stem cells by defined factors, Cell Stem Cell, 10, 465, 10.1016/j.stem.2012.02.021

Hanna, 2009, Direct cell reprogramming is a stochastic process amenable to acceleration, Nature, 462, 595, 10.1038/nature08592

Hirai, 2012, Efficient iPS cell production with the MyoD transactivation domain in serum-free culture, PLoS One, 7, e34149, 10.1371/journal.pone.0034149

Hirami, 2009, Generation of retinal cells from mouse and human induced pluripotent stem cells, Neurosci. Lett., 458, 126, 10.1016/j.neulet.2009.04.035

Howden, 2011, Genetic correction and analysis of induced pluripotent stem cells from a patient with gyrate atrophy, Proc. Natl. Acad. Sci. U. S. A., 108, 6537, 10.1073/pnas.1103388108

Hu, 2010, Memory in induced pluripotent stem cells: reprogrammed human retinal-pigmented epithelial cells show tendency for spontaneous redifferentiation, Stem Cells, 28, 1981, 10.1002/stem.531

Hu, 2012, A novel approach for subretinal implantation of ultrathin substrates containing stem cell-derived retinal pigment epithelium monolayer, Ophthalmic Res., 48, 186, 10.1159/000338749

Huangfu, 2008, Induction of pluripotent stem cells by defined factors is greatly improved by small-molecule compounds, Nat. Biotechnol., 26, 795, 10.1038/nbt1418

Idelson, 2009, Directed differentiation of human embryonic stem cells into functional retinal pigment epithelium cells, Cell Stem Cell, 5, 396, 10.1016/j.stem.2009.07.002

Ieda, 2010, Direct reprogramming of fibroblasts into functional cardiomyocytes by defined factors, Cell, 142, 375, 10.1016/j.cell.2010.07.002

Inoue, 2007, Subretinal transplantation of bone marrow mesenchymal stem cells delays retinal degeneration in the RCS rat model of retinal degeneration, Exp. Eye Res., 85, 234, 10.1016/j.exer.2007.04.007

Itskovitz-Eldor, 2000, Differentiation of human embryonic stem cells into embryoid bodies compromising the three embryonic germ layers, Mol. Med., 6, 88, 10.1007/BF03401776

Itzhaki, 2011, Modelling the long QT syndrome with induced pluripotent stem cells, Nature, 471, 225, 10.1038/nature09747

Jaenisch, 2008, Stem cells, the molecular circuitry of pluripotency and nuclear reprogramming, Cell, 132, 567, 10.1016/j.cell.2008.01.015

Jin, 2011, Modeling retinal degeneration using patient-specific induced pluripotent stem cells, PLoS One, 6, e17084, 10.1371/journal.pone.0017084

Judson, 2009, Embryonic stem cell-specific microRNAs promote induced pluripotency, Nat. Biotechnol., 27, 459, 10.1038/nbt.1535

Kaji, 2009, Virus-free induction of pluripotency and subsequent excision of reprogramming factors, Nature, 458, 771, 10.1038/nature07864

Kamiya, 2011, Intrinsic transition of embryonic stem-cell differentiation into neural progenitors, Nature, 470, 503, 10.1038/nature09726

Kim, 2009, Generation of human induced pluripotent stem cells by direct delivery of reprogramming proteins, Cell Stem Cell, 4, 472, 10.1016/j.stem.2009.05.005

Kim, 2010, Epigenetic memory in induced pluripotent stem cells, Nature, 467, 285, 10.1038/nature09342

Klimanskaya, 2004, Derivation and comparative assessment of retinal pigment epithelium from human embryonic stem cells using transcriptomics, Cloning Stem Cells, 6, 217, 10.1089/clo.2004.6.217

Kokkinaki, 2011, Human induced pluripotent stem-derived retinal pigment epithelium (RPE) cells exhibit ion transport, membrane potential, polarized vascular endothelial growth factor secretion, and gene expression pattern similar to native RPE, Stem Cells, 29, 825, 10.1002/stem.635

Koso, 2009, CD73, a novel cell surface antigen that characterizes retinal photoreceptor precursor cells, Invest. Ophthalmol. Vis. Sci., 50, 5411, 10.1167/iovs.08-3246

Ku, 2010, Friedreich's ataxia induced pluripotent stem cells model intergenerational GAATTC triplet repeat instability, Cell Stem Cell, 7, 631, 10.1016/j.stem.2010.09.014

Kurosawa, 2007, Methods for inducing embryoid body formation: in vitro differentiation system of embryonic stem cells, J. Biosci. Bioeng., 103, 389, 10.1263/jbb.103.389

Kuzmenkin, 2009, Functional characterization of cardiomyocytes derived from murine induced pluripotent stem cells in vitro, FASEB J., 23, 4168, 10.1096/fj.08-128546

Lakowski, 2010, Cone and rod photoreceptor transplantation in models of the childhood retinopathy Leber congenital amaurosis using flow-sorted Crx-positive donor cells, Hum. Mol. Genet., 19, 4545, 10.1093/hmg/ddq378

Lakowski, 2011, Effective transplantation of photoreceptor precursor cells selected via cell surface antigen expression, Stem Cells, 29, 1391, 10.1002/stem.694

Lamba, 2006, Efficient generation of retinal progenitor cells from human embryonic stem cells, Proc. Natl. Acad. Sci. U. S. A., 103, 12769, 10.1073/pnas.0601990103

Lamba, 2009, Transplantation of human embryonic stem cell-derived photoreceptors restores some visual function in Crx-deficient mice, Cell Stem Cell, 4, 73, 10.1016/j.stem.2008.10.015

Lamba, 2010, Generation, purification and transplantation of photoreceptors derived from human induced pluripotent stem cells, PLoS One, 5, e8763, 10.1371/journal.pone.0008763

Laurent, 2011, 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, 8, 106, 10.1016/j.stem.2010.12.003

Lee, 2007, Directed differentiation and transplantation of human embryonic stem cell-derived motoneurons, Stem Cells, 25, 1931, 10.1634/stemcells.2007-0097

Li, 2012, Long-term safety and efficacy of human-induced pluripotent stem cell (iPS) grafts in a preclinical model of retinitis pigmentosa, Mol. Med., 18, 1312, 10.2119/molmed.2012.00242

Liao, 2010, Molecular signature of primary retinal pigment epithelium and stem-cell-derived RPE cells, Hum. Mol. Genet., 19, 4229, 10.1093/hmg/ddq341

Lin, 2009, A chemical platform for improved induction of human iPSCs, Nat. Methods, 6, 805, 10.1038/nmeth.1393

Lowry, 2008, Generation of human induced pluripotent stem cells from dermal fibroblasts, Proc. Natl. Acad. Sci. U. S. A., 105, 2883, 10.1073/pnas.0711983105

Lu, 2009, Long-term safety and function of RPE from human embryonic stem cells in preclinical models of macular degeneration, Stem Cells, 27, 2126, 10.1002/stem.149

Lujan, 2010, An imprinted signature helps isolate ESC-equivalent iPSCs, Cell Res., 20, 974, 10.1038/cr.2010.117

Lujan, 2012, Direct conversion of mouse fibroblasts to self-renewing, tripotent neural precursor cells, Proc. Natl. Acad. Sci. U. S. A., 109, 2527, 10.1073/pnas.1121003109

MacLaren, 2006, Retinal repair by transplantation of photoreceptor precursors, Nature, 444, 203, 10.1038/nature05161

Mandai, 2010, Use of lectins to enrich mouse ES-derived retinal progenitor cells for the purpose of transplantation therapy, Cell Transplant., 19, 9, 10.3727/096368909X476599

Marchetto, 2010, A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells, Cell, 143, 527, 10.1016/j.cell.2010.10.016

Mattis, 2012, Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes, Cell Stem Cell, 11, 264, 10.1016/j.stem.2012.04.027

McGill, 2007, Syngeneic Schwann cell transplantation preserves vision in RCS rat without immunosuppression, Invest. Ophthalmol. Vis. Sci., 48, 1906, 10.1167/iovs.06-1117

McLellan, 2003, NIH director reviews first year on the job. “Roadmap” calls for reorganisation of basic and clinical research, Lancet, 362, 381, 10.1016/S0140-6736(03)14064-0

McUsic, 2012, Guiding the morphogenesis of dissociated newborn mouse retinal cells and hES cell-derived retinal cells by soft lithography-patterned microchannel PLGA scaffolds, Biomaterials, 33, 1396, 10.1016/j.biomaterials.2011.10.083

Mekhoubad, 2012, Erosion of dosage compensation impacts human iPSC disease modeling, Cell Stem Cell, 10, 595, 10.1016/j.stem.2012.02.014

Mellough, 2012, Efficient stage-specific differentiation of human pluripotent stem cells toward retinal photoreceptor cells, Stem Cells, 30, 673, 10.1002/stem.1037

Meyer, 2009, Modeling early retinal development with human embryonic and induced pluripotent stem cells, Proc. Natl. Acad. Sci. U. S. A., 106, 16698, 10.1073/pnas.0905245106

Meyer, 2011, Optic vesicle-like structures derived from human pluripotent stem cells facilitate a customized approach to retinal disease treatment, Stem Cells, 29, 1206, 10.1002/stem.674

Mikkelsen, 2008, Dissecting direct reprogramming through integrative genomic analysis, Nature, 454, 49, 10.1038/nature07056

Moretti, 2010, Patient-specific induced pluripotent stem-cell models for long-QT syndrome, N. Engl. J. Med., 363, 1397, 10.1056/NEJMoa0908679

MRC, 2013

Munoz-Sanjuan, 2002, Neural induction, the default model and embryonic stem cells, Nat. Rev. Neurosci., 3, 271, 10.1038/nrn786

Nakanishi, 2012, Development of Sendai virus vectors and their potential applications in gene therapy and regenerative medicine, Curr. Gene Ther., 12, 410, 10.2174/156652312802762518

Nakano, 2012, Self-formation of optic cups and storable stratified neural retina from human ESCs, Cell Stem Cell, 10, 771, 10.1016/j.stem.2012.05.009

Nasonkin, 2013, Conditional knockdown of DNA methyltransferase 1 reveals a key role of retinal pigment epithelium integrity in photoreceptor outer segment morphogenesis, Development, 140, 1330, 10.1242/dev.086603

Nguyen, 2000, Signaling and transcriptional regulation in early mammalian eye development: a link between FGF and MITF, Development, 127, 3581, 10.1242/dev.127.16.3581

Okita, 2011, Immunogenicity of induced pluripotent stem cells, Circ. Res., 109, 720, 10.1161/RES.0b013e318232e187

Okita, 2008, Generation of mouse induced pluripotent stem cells without viral vectors, Science, 322, 949, 10.1126/science.1164270

Osakada, 2008, Toward the generation of rod and cone photoreceptors from mouse, monkey and human embryonic stem cells, Nat. Biotechnol., 26, 215, 10.1038/nbt1384

Osakada, 2009, In vitro differentiation of retinal cells from human pluripotent stem cells by small-molecule induction, J. Cell Sci., 122, 3169, 10.1242/jcs.050393

Pearson, 2010, Targeted disruption of outer limiting membrane junctional proteins (Crb1 and ZO-1) increases integration of transplanted photoreceptor precursors into the adult wild-type and degenerating retina, Cell Transplant., 19, 487, 10.3727/096368909X486057

Pearson, 2012, Restoration of vision after transplantation of photoreceptors, Nature, 485, 99, 10.1038/nature10997

Peng, 2010, The action potential and comparative pharmacology of stem cell-derived human cardiomyocytes, J. Pharmacol. Toxicol. Methods, 61, 277, 10.1016/j.vascn.2010.01.014

Phillips, 2012, Blood-derived human iPS cells generate optic vesicle-like structures with the capacity to form retinal laminae and develop synapses, Invest. Ophthalmol. Vis. Sci., 53, 2007, 10.1167/iovs.11-9313

Pomp, 2011, Unexpected X chromosome skewing during culture and reprogramming of human somatic cells can be alleviated by exogenous telomerase, Cell Stem Cell, 9, 156, 10.1016/j.stem.2011.06.004

Prather, 1989, Nuclear transplantation in early pig embryos, Biol. Reprod., 41, 414, 10.1095/biolreprod41.3.414

Ramalho-Santos, 2011, Human procreation in unchartered territory: new twists in ethical discussions, Hum. Reprod., 26, 1284, 10.1093/humrep/der093

Reubinoff, 2001, Neural progenitors from human embryonic stem cells, Nat. Biotechnol., 19, 1134, 10.1038/nbt1201-1134

Robertson, 2007, Embryonic stem cell-derived tissues are immunogenic but their inherent immune privilege promotes the induction of tolerance, Proc. Natl. Acad. Sci. U. S. A., 104, 20920, 10.1073/pnas.0710265105

Rowland, 2012, Pluripotent human stem cells for the treatment of retinal disease, J. Cell. Physiol., 227, 457, 10.1002/jcp.22814

Schwartz, 2012, Embryonic stem cell trials for macular degeneration: a preliminary report, Lancet, 379, 713, 10.1016/S0140-6736(12)60028-2

Sheng, 2012, Direct reprogramming of Sertoli cells into multipotent neural stem cells by defined factors, Cell Res., 22, 208, 10.1038/cr.2011.175

Sheridan, 2011, Epigenetic characterization of the FMR1 gene and aberrant neurodevelopment in human induced pluripotent stem cell models of fragile X syndrome, PLoS One, 6, e26203, 10.1371/journal.pone.0026203

Singh, 2013, iPS cell modeling of Best disease: insights into the pathophysiology of an inherited macular degeneration, Hum. Mol. Genet., 22, 593, 10.1093/hmg/dds469

Si-Tayeb, 2010, Generation of human induced pluripotent stem cells by simple transient transfection of plasmid DNA encoding reprogramming factors, BMC Dev. Biol., 10, 81, 10.1186/1471-213X-10-81

Smith, 2008, Inhibition of Activin/Nodal signaling promotes specification of human embryonic stem cells into neuroectoderm, Dev. Biol., 313, 107, 10.1016/j.ydbio.2007.10.003

Smukler, 2006, Embryonic stem cells assume a primitive neural stem cell fate in the absence of extrinsic influences, J. Cell Biol., 172, 79, 10.1083/jcb.200508085

Soldner, 2009, Parkinson's disease patient-derived induced pluripotent stem cells free of viral reprogramming factors, Cell, 136, 964, 10.1016/j.cell.2009.02.013

Somers, 2010, Generation of transgene-free lung disease-specific human induced pluripotent stem cells using a single excisable lentiviral stem cell cassette, Stem Cells, 28, 1728, 10.1002/stem.495

Sonoda, 2009, A protocol for the culture and differentiation of highly polarized human retinal pigment epithelial cells, Nat. Protoc., 4, 662, 10.1038/nprot.2009.33

Sridhar, 2013, Nonxenogeneic growth and retinal differentiation of human induced pluripotent stem cells, Stem Cells Transl. Med., 2, 255, 10.5966/sctm.2012-0101

Stacpoole, 2011, Derivation of neural precursor cells from human ES cells at 3% O(2) is efficient, enhances survival and presents no barrier to regional specification and functional differentiation, Cell Death Differ., 18, 1016, 10.1038/cdd.2010.171

Stadtfeld, 2012, Ascorbic acid prevents loss of Dlk1-Dio3 imprinting and facilitates generation of all-iPS cell mice from terminally differentiated B cells, Nat Genet, 44, 10.1038/ng0712-831b

Stadtfeld, 2008, Induced pluripotent stem cells generated without viral integration, Science, 322, 945, 10.1126/science.1162494

Strunnikova, 2010, Transcriptome analysis and molecular signature of human retinal pigment epithelium, Hum. Mol. Genet., 19, 2468, 10.1093/hmg/ddq129

Szabo, 2010, Direct conversion of human fibroblasts to multilineage blood progenitors, Nature, 468, 521, 10.1038/nature09591

Takahashi, 2006, Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors, Cell, 126, 663, 10.1016/j.cell.2006.07.024

Takahashi, 2007, Induction of pluripotent stem cells from adult human fibroblasts by defined factors, Cell, 131, 861, 10.1016/j.cell.2007.11.019

Tchieu, 2010, Female human iPSCs retain an inactive X chromosome, Cell Stem Cell, 7, 329, 10.1016/j.stem.2010.06.024

Thier, 2012, Direct conversion of fibroblasts into stably expandable neural stem cells, Cell Stem Cell, 10, 473, 10.1016/j.stem.2012.03.003

Thomson, 1998, Embryonic stem cell lines derived from human blastocysts, Science, 282, 1145, 10.1126/science.282.5391.1145

Tropepe, 2001, Direct neural fate specification from embryonic stem cells: a primitive mammalian neural stem cell stage acquired through a default mechanism, Neuron, 30, 65, 10.1016/S0896-6273(01)00263-X

Tucker, 2011, Exome sequencing and analysis of induced pluripotent stem cells identify the cilia-related gene male germ cell-associated kinase (MAK) as a cause of retinitis pigmentosa, Proc. Natl. Acad. Sci. U. S. A., 108, E569, 10.1073/pnas.1108918108

Tucker, 2013, Use of a synthetic xeno-free culture substrate for induced pluripotent stem cell induction and retinal differentiation, Stem Cells Transl. Med., 2, 16, 10.5966/sctm.2012-0040

Ukrohne, 2012, Generation of retinal pigment epithelial cells from small molecules and OCT4 reprogrammed human induced pluripotent stem cells, Stem Cells Transl. Med., 1, 96, 10.5966/sctm.2011-0057

Urbach, 2010, Differential modeling of fragile X syndrome by human embryonic stem cells and induced pluripotent stem cells, Cell Stem Cell, 6, 407, 10.1016/j.stem.2010.04.005

Utikal, 2009, Sox2 is dispensable for the reprogramming of melanocytes and melanoma cells into induced pluripotent stem cells, J. Cell Sci., 122, 3502, 10.1242/jcs.054783

Vaajasaari, 2011, Toward the defined and xeno-free differentiation of functional human pluripotent stem cell-derived retinal pigment epithelial cells, Mol. Vis., 17, 558

Vallier, 2005, Activin/Nodal and FGF pathways cooperate to maintain pluripotency of human embryonic stem cells, J. Cell Sci., 118, 4495, 10.1242/jcs.02553

Vierbuchen, 2010, Direct conversion of fibroblasts to functional neurons by defined factors, Nature, 463, 1035, 10.1038/nature08797

Vugler, 2008, Elucidating the phenomenon of HESC-derived RPE: anatomy of cell genesis, expansion and retinal transplantation, Exp. Neurol., 214, 347, 10.1016/j.expneurol.2008.09.007

Wang, 2008, Long-term vision rescue by human neural progenitors in a rat model of photoreceptor degeneration, Invest. Ophthalmol. Vis. Sci., 49, 3201, 10.1167/iovs.08-1831

Wang, 2011, The histone demethylases Jhdm1a/1b enhance somatic cell reprogramming in a vitamin-C-dependent manner, Cell Stem Cell, 9, 575, 10.1016/j.stem.2011.10.005

Warren, 2010, Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA, Cell Stem Cell, 7, 618, 10.1016/j.stem.2010.08.012

Wernig, 2008, A drug-inducible transgenic system for direct reprogramming of multiple somatic cell types, Nat. Biotechnol., 26, 916, 10.1038/nbt1483

West, 2008, Pharmacological disruption of the outer limiting membrane leads to increased retinal integration of transplanted photoreceptor precursors, Exp. Eye Res., 86, 601, 10.1016/j.exer.2008.01.004

West, 2010, Long-term survival of photoreceptors transplanted into the adult murine neural retina requires immune modulation, Stem Cells, 28, 1997, 10.1002/stem.520

West, 2012, Defining the integration capacity of embryonic stem cell-derived photoreceptor precursors, Stem Cells, 30, 1424, 10.1002/stem.1123

Williams, 2011, The many different cellular functions of MYO7A in the retina, Biochem. Soc. Trans., 39, 1207, 10.1042/BST0391207

Wilmut, 1997, Viable offspring derived from fetal and adult mammalian cells, Nature, 385, 810, 10.1038/385810a0

Woltjen, 2009, piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells, Nature, 458, 766, 10.1038/nature07863

Yu, 2007, Induced pluripotent stem cell lines derived from human somatic cells, Science, 318, 1917, 10.1126/science.1151526

Yusa, 2011, Targeted gene correction of alpha1-antitrypsin deficiency in induced pluripotent stem cells, Nature, 478, 391, 10.1038/nature10424

Zhang, 2010, CD73: a novel target for cancer immunotherapy, Cancer Res., 70, 6407, 10.1158/0008-5472.CAN-10-1544

Zhang, 2013, Direct conversion of human fibroblasts into retinal pigment epithelium-like cells by defined factors, Protein Cell, 10.1007/s13238-013-0011-2

Zhao, 2011, Immunogenicity of induced pluripotent stem cells, Nature, 474, 212, 10.1038/nature10135

Zhou, 2009, Generation of induced pluripotent stem cells using recombinant proteins, Cell Stem Cell, 4, 381, 10.1016/j.stem.2009.04.005

Zhou, 2011, Differentiation of induced pluripotent stem cells of swine into rod photoreceptors and their integration into the retina, Stem Cells, 29, 972, 10.1002/stem.637

Zhou, 2009, Adenoviral gene delivery can reprogram human fibroblasts to induced pluripotent stem cells, Stem Cells, 27, 2667, 10.1002/stem.201

Zou, 2009, Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells, Cell Stem Cell, 5, 97, 10.1016/j.stem.2009.05.023