Induced pluripotent stem cell technology: a decade of progress.

Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors

Takahashi, K., & Yamanaka, S. (2006) · Cell · doi:10.1016/j.cell.2006.07.024 · Cited by 26,673

Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors

Takahashi, K., Tanabe, K., Ohnuki, M., Narita, M., Ichisaka, T., Tomoda, K., & Yamanaka, S. (2007) · Cell · doi:10.1016/j.cell.2007.11.019 · Cited by 20,221

A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity

Jinek, M., Chylinski, K., Fonfara, I., Hauer, M., Doudna, J. A., & Charpentier, E. (2012) · Science · doi:10.1126/science.1225829 · Cited by 17,116

Embryonic Stem Cell Lines Derived from Human Blastocysts

Thomson, J. A., Itskovitz‐Eldor, J., Shapiro, S. S., Waknitz, M. A., Swiergiel, J. J., Marshall, V. S., & Jones, J. M. (1998) · Science · doi:10.1126/science.282.5391.1145 · Cited by 15,947

Multiplex Genome Engineering Using CRISPR/Cas Systems

Cong, L., Ran, F. A., Cox, D., Lin, S., Barretto, R., Habib, N., Hsu, P. D., Wu, X., Jiang, W., Marraffini, L. A., et al. (2013) · Science · doi:10.1126/science.1231143 · Cited by 15,339

Induced Pluripotent Stem Cell Lines Derived from Human Somatic Cells

Yu, J., Vodyanik, M. A., Smuga-Otto, K., Antosiewicz-Bourget, J., Frane, J. L., Tian, S., Nie, J., Jonsdottir, G. A., Ruotti, V., Stewart, R., et al. (2007) · Science · doi:10.1126/science.1151526 · Cited by 10,310

RNA-Guided Human Genome Engineering via Cas9

Mali, P., Yang, L., Esvelt, K. M., Aach, J., Guell, M., DiCarlo, J. E., Norville, J. E., & Church, G. M. (2013) · Science · doi:10.1126/science.1232033 · Cited by 9,210

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

Komor, A. C., Kim, Y. B., Packer, M. S., Zuris, J. A., & Liu, D. R. (2016) · Nature · doi:10.1038/nature17946 · Cited by 5,437

Cerebral organoids model human brain development and microcephaly

Lancaster, M. A., Renner, M., Martin, C.-A., Wenzel, D., Bicknell, L. S., Hurles, M. E., Homfray, T., Penninger, J. M., Jackson, A. P., & Knoblich, J. A. (2013) · Nature · doi:10.1038/nature12517 · Cited by 5,372

Genome-scale CRISPR-Cas9 knockout screening in human cells

Shalem, O., Sanjana, N. E., Hartenian, E., Shi, X., Scott, D. A., Mikkelsen, T. S., Heckl, D., Ebert, B. L., Root, D. E., Doench, J. G., et al. (2014) · Science · doi:10.1126/science.1247005 · Cited by 5,234

Organoid Models and Applications in Biomedical Research

Xinaris, C., Brizi, V., & Remuzzi, G. (2015) · The Nephron journals/Nephron journals · doi:10.1159/000433566 · Cited by 3,519

Innovation in the pharmaceutical industry: New estimates of R&D costs

DiMasi, J. A., Grabowski, H. G., & Hansen, R. W. (2016) · Journal of Health Economics · doi:10.1016/j.jhealeco.2016.01.012 · Cited by 3,359

Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity

Ran, F. A., Hsu, P. D., Lin, C.-Y., Gootenberg, J. S., Konermann, S., Trevino, A. E., Scott, D. A., Inoue, A., Matoba, S., Zhang, Y., et al. (2013) · Cell · doi:10.1016/j.cell.2013.08.021 · Cited by 3,299

High-Frequency off-Target Mutagenesis Induced by CRISPR-Cas Nucleases in Human Cells

Fu, Y., Foden, J. A., Khayter, C., Maeder, M. L., Reyon, D., Joung, J. K., & Sander, J. D. (2013) · Nature Biotechnology · doi:10.1038/nbt.2623 · Cited by 3,292

Organogenesis in a dish: Modeling development and disease using organoid technologies

Lancaster, M. A., & Knoblich, J. A. (2014) · Science · doi:10.1126/science.1247125 · Cited by 2,931

Highly efficient reprogramming to pluripotency and directed differentiation of human cells with synthetic modified mRNA

Warren, L., Manos, P. D., Ahfeldt, T., Loh, Y.-H., Li, H., Lau, F., Ebina, W., Mandal, P. K., Smith, Z. D., Meissner, A., et al. (2010) · Cell Stem Cell · doi:10.1016/j.stem.2010.08.012 · Cited by 2,662

Rationally engineered Cas9 nucleases with improved specificity

Slaymaker, I. M., Gao, L., Zetsche, B., Scott, D. A., Yan, W. X., & Zhang, F. (2016) · Science · doi:10.1126/science.aad5227 · Cited by 2,414

Human induced pluripotent stem cells free of vector and transgene sequences

Yu, J., Hu, K., Smuga-Otto, K., Tian, S., Stewart, R., Slukvin, I. I., & Thomson, J. A. (2009) · Science · doi:10.1126/science.1172482 · Cited by 2,386

Cas-OFFinder: A fast and versatile algorithm that searches for potential off-target sites of Cas9 RNA-guided endonucleases

Bae, S., Park, J., & Kim, J.-S. (2014) · Bioinformatics · doi:10.1093/bioinformatics/btu048 · Cited by 2,339

Vascularized and functional human liver from an iPSC-derived organ bud transplant

Takebe, T., Sekine, K., Enomura, M., Koike, H., Kimura, M., Ogaeri, T., Zhang, R.-R., Ueno, Y., Zheng, Y.-W., Koike, N., et al. (2013) · Nature · doi:10.1038/nature12271 · Cited by 2,170

Brain-Region-Specific Organoids Using Mini-bioreactors for Modeling ZIKV Exposure

Qian, X., Nguyen, H. N., Song, M. M., Hadiono, C., Ogden, S. C., Hammack, C., Yao, B., Hamersky, G. R., Jacob, F., Zhong, C., et al. (2016) · Cell · doi:10.1016/j.cell.2016.04.032 · Cited by 2,127

RNA-programmed genome editing in human cells

Jinek, M., East, A., Cheng, A., Lin, S., Ma, E., & Doudna, J. (2013) · eLife · doi:10.7554/elife.00471 · Cited by 2,098

A more efficient method to generate integration-free human iPS cells

Okita, K., Matsumura, Y., Sato, Y., Okada, A., Morizane, A., Okamoto, S., Hong, H., Nakagawa, M., Tanabe, K., Tezuka, K.-I., et al. (2011) · Nature Methods · doi:10.1038/nmeth.1591 · Cited by 2,061

Dynamic Imaging of Genomic Loci in Living Human Cells by an Optimized CRISPR/Cas System

Chen, B., Gilbert, L. A., Cimini, B. A., Schnitzbauer, J., Zhang, W., Li, G., Park, J. S., Blackburn, E. H., Weissman, J. S., Qi, L. S., et al. (2013) · Cell · doi:10.1016/j.cell.2013.12.001 · Cited by 1,988

Directed differentiation of human pluripotent stem cells into intestinal tissue in vitro

Spence, J. R., Mayhew, C. N., Rankin, S. A., Kuhar, M. F., Vallance, J. E., Tolle, K. A., Hoskins, E. E., Kalinichenko, V. V., Wells, S. I., Zorn, A. M., et al. (2010) · Nature · doi:10.1038/nature09691 · Cited by 1,911

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

Christian, M., Cermak, T., Doyle, E. L., Schmidt, C., Zhang, F., Hummel, A., Bogdanove, A. J., & Voytas, D. F. (2010) · Genetics · doi:10.1534/genetics.110.120717 · Cited by 1,869

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

Cho, S. W., Kim, S., Kim, J. M., & Kim, J.-S. (2013) · Nature Biotechnology · doi:10.1038/nbt.2507 · Cited by 1,863

Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins

Kim, D., Kim, C.-H., Moon, J.-I., Chung, Y.-G., Chang, M.-Y., Han, B.-S., Ko, S., Yang, E., Cha, K. Y., Lanza, R., et al. (2009) · Cell stem cell · doi:10.1016/j.stem.2009.05.005 · Cited by 1,814

piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells

Woltjen, K., Michael, I. P., Mohseni, P., Desai, R., Mileikovsky, M., Hämäläinen, R. H., Cowling, R. A., Wang, W., Liu, P., Gertsenstein, M., et al. (2009) · Nature · doi:10.1038/nature07863 · Cited by 1,810

Induced pluripotent stem cells generated without viral integration

Stadtfeld, M., Nagaya, M., Utikal, J., Weir, G., & Hochedlinger, K. (2008) · Science · doi:10.1126/science.1162494 · Cited by 1,683

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

Urnov, F. D., Miller, J. C., Lee, Y.-L., Beausejour, C. M., Rock, J. M., Augustus, S., Jamieson, A. C., Porteus, M. H., Gregory, P. D., & Holmes, M. C. (2005) · Nature · doi:10.1038/nature03556 · Cited by 1,642

Kidney organoids from human iPS cells contain multiple lineages and model human nephrogenesis

Takasato, M., Er, P. X., Chiu, H. S., Maier, B., Baillie, G. J., Ferguson, C., Parton, R. G., Wolvetang, E. J., Roost, M. S., Chuva de Sousa Lopes, S. M., et al. (2015) · Nature · doi:10.1038/nature15695 · Cited by 1,627

Functional cortical neurons and astrocytes from human pluripotent stem cells in 3D culture

Paşca, A. M., Sloan, S. A., Clarke, L. E., Tian, Y., Makinson, C. D., Huber, N., Kim, C. H., Park, J.-Y., O'Rourke, N. A., Nguyen, K. D., et al. (2015) · Nature Methods · doi:10.1038/nmeth.3415 · Cited by 1,609

Treatment of Sickle Cell Anemia Mouse Model with iPS Cells Generated from Autologous Skin

Hanna, J. H., Wernig, M., Markoulaki, S., Sun, C., Meissner, A., Cassady, J. P., Beard, C., Brambrink, T., Wu, L.-C., Townes, T. M., et al. (2007) · Science · doi:10.1126/science.1152092 · Cited by 1,521

Organoids as an in vitro model of human development and disease

Fatehullah, A., Tan, S. H., & Barker, N. (2016) · Nature Cell Biology · doi:10.1038/ncb3312 · Cited by 1,508

Immunogenicity of induced pluripotent stem cells

Zhao, T., Zhang, Z., Rong, Z., & Xu, Y. (2011) · Nature · doi:10.1038/nature10135 · Cited by 1,484

Astroglia induce neurogenesis from adult neural stem cells

Song, H., Stevens, C. F., & Gage, F. H. (2002) · Nature · doi:10.1038/417039a · Cited by 1,452

Self-formation of optic cups and storable stratified neural retina from human ESCs

Nakano, T., Ando, S., Takata, N., Kawada, M., Muguruma, K., Sekiguchi, K., Saito, K., Yonemura, S., Eiraku, M., & Sasai, Y. (2012) · Cell stem cell · doi:10.1016/j.stem.2012.05.009 · Cited by 1,447

Induced pluripotent stem cells from a spinal muscular atrophy patient

Ebert, A. D., Yu, J., Rose, F. F., Mattis, V. B., Lorson, C. L., Thomson, J. A., & Svendsen, C. N. (2009) · Nature · doi:10.1038/nature07677 · Cited by 1,441

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

FUSAKI, N., BAN, H., NISHIYAMA, A., SAEKI, K., & HASEGAWA, M. (2009) · Proceedings of the Japan Academy Series B · doi:10.2183/pjab.85.348 · Cited by 1,411

Pluripotent stem cells induced from mouse somatic cells by small-molecule compounds.

Hou, P., Li, Y., Zhang, X., Liu, C., Guan, J., Li, H., Zhao, T., Ye, J., Yang, W., Liu, K., et al. (2013) · Science · doi:10.1126/science.1239278 · Cited by 1,402

Modelling schizophrenia using human induced pluripotent stem cells

Brennand, K. J., Simone, A., Jou, J., Gelboin-Burkhart, C., Tran, N., Sangar, S., Li, Y., Mu, Y., Chen, G., Yu, D., et al. (2011) · Nature · doi:10.1038/nature09915 · Cited by 1,375

RNA-guided gene activation by CRISPR-Cas9-based transcription factors

Perez-Pinera, P., Kocak, D. D., Vockley, C. M., Adler, A. F., Kabadi, A. M., Polstein, L. R., Thakore, P. I., Glass, K. A., Ousterout, D. G., Leong, K. W., et al. (2013) · Nature Methods · doi:10.1038/nmeth.2600 · Cited by 1,328

The Brazilian Zika virus strain causes birth defects in experimental models

Cugola, F. R., Fernandes, I. R., Russo, F. B., Freitas, B. C., Dias, J. L. M., Guimarães, K. P., Benazzato, C., Almeida, N., Pignatari, G. C., Romero, S., et al. (2016) · Nature · doi:10.1038/nature18296 · Cited by 1,326

Probing sporadic and familial Alzheimer’s disease using induced pluripotent stem cells

Israel, M. A., Yuan, S. H., Bardy, C., Reyna, S. M., Mu, Y., Herrera, C., Hefferan, M. P., Van Gorp, S., Nazor, K. L., Boscolo, F. S., et al. (2012) · Nature · doi:10.1038/nature10821 · Cited by 1,253

Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons

Nagai, M., Re, D. B., Nagata, T., Chalazonitis, A., Jessell, T. M., Wichterle, H., & Przedborski, S. (2007) · Nature Neuroscience · doi:10.1038/nn1876 · Cited by 1,220

Zika virus impairs growth in human neurospheres and brain organoids

Garcez, P. P., Loiola, E. C., Madeiro da Costa, R., Higa, L. M., Trindade, P., Delvecchio, R., Nascimento, J. M., Brindeiro, R., Tanuri, A., & Rehen, S. K. (2016) · Science · doi:10.1126/science.aaf6116 · Cited by 1,184

Genetic engineering of human pluripotent cells using TALE nucleases

Hockemeyer, D. F., Wang, H., Kiani, S., Lai, C. S., Gao, Q., Cassady, J. P., Cost, G. J., Zhang, L., Santiago, Y., Miller, J. C., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1927 · Cited by 1,161

FOXG1-Dependent Dysregulation of GABA/Glutamate Neuron Differentiation in Autism Spectrum Disorders

Mariani, J., Coppola, G., Zhang, P., Abyzov, A., Provini, L., Tomasini, L., Amenduni, M., Szekely, A., Palejev, D., Wilson, M., et al. (2015) · Cell · doi:10.1016/j.cell.2015.06.034 · Cited by 1,149

Human cerebral organoids recapitulate gene expression programs of fetal neocortex development

Camp, J. G., Badsha, F., Florio, M., Kanton, S., Gerber, T., Wilsch-Bräuninger, M., Lewitus, E., Sykes, A., Hevers, W., Lancaster, M., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1520760112 · Cited by 1,130

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

Hockemeyer, D. F., Soldner, F., Beard, C., Gao, Q., Mitalipova, M. M., DeKelver, R. C., Katibah, G. E., Amora, R., Boydston, E. A., Zeitler, B., et al. (2009) · Nature Biotechnology · doi:10.1038/nbt.1562 · Cited by 1,064

Genome-wide recessive genetic screening in mammalian cells with a lentiviral CRISPR-guide RNA library

Koike-Yusa, H., Li, Y., Tan, E.-P., Velasco-Herrera, M. D. C., & Yusa, K. (2014) · Nature Biotechnology · doi:10.1038/nbt.2800 · Cited by 1,064

Self-organization of axial polarity, inside-out layer pattern, and species-specific progenitor dynamics in human ES cell–derived neocortex

Kadoshima, T., Sakaguchi, H., Nakano, T., Soen, M., Ando, S., Eiraku, M., & Sasai, Y. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1315710110 · Cited by 999

Modelling human development and disease in pluripotent stem-cell-derived gastric organoids

McCracken, K. W., Catá, E. M., Crawford, C. M., Sinagoga, K. L., Schumacher, M., Rockich, B. E., Tsai, Y.-H., Mayhew, C. N., Spence, J. R., Zavros, Y., et al. (2014) · Nature · doi:10.1038/nature13863 · Cited by 980

Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9

Paquet, D., Kwart, D., Chen, A., Sproul, A., Jacob, S., Teo, S., Olsen, K. M., Gregg, A., Noggle, S., & Tessier-Lavigne, M. (2016) · Nature · doi:10.1038/nature17664 · Cited by 951

A decade of transcription factor-mediated reprogramming to pluripotency

Takahashi, K., & Yamanaka, S. (2016) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm.2016.8 · Cited by 894

Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs

Lee, G., Papapetrou, E. P., Kim, H., Chambers, S. M., Tomishima, M. J., Fasano, C. A., Ganat, Y. M., Menon, J., Shimizu, F., Viale, A. J., et al. (2009) · Nature · doi:10.1038/nature08320 · Cited by 893

Variation in the safety of induced pluripotent stem cell lines

Miura, K., Okada, Y., Aoi, T., Okada, A., Takahashi, K., Okita, K., Nakagawa, M., Koyanagi, M., Tanabe, K., Ohnuki, M., et al. (2009) · Nature Biotechnology · doi:10.1038/nbt.1554 · Cited by 891

Induced Pluripotent Stem Cells: Past, Present, and Future

Yamanaka, S. (2012) · Cell stem cell · doi:10.1016/j.stem.2012.05.005 · Cited by 878

Engineering Stem Cell Organoids

Yin, X., Mead, B. E., Safaee, H., Langer, R., Karp, J. M., & Levy, O. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2015.12.005 · Cited by 866

In vitro generation of human pluripotent stem cell derived lung organoids

Dye, B. R., Hill, D. R., Ferguson, M. A. H., Tsai, Y.-H., Nagy, M. S., Dyal, R., Wells, J. M., Mayhew, C. N., Nattiv, R., Klein, O. D., et al. (2015) · eLife · doi:10.7554/elife.05098 · Cited by 846

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

Lombardo, A., Genovese, P., Beausejour, C. M., Colleoni, S., Lee, Y.-L., Kim, K. A., Ando, D., Urnov, F. D., Galli, C., Gregory, P. D., et al. (2007) · Nature Biotechnology · doi:10.1038/nbt1353 · Cited by 845

Beyond editing: repurposing CRISPR-Cas9 for precision genome regulation and interrogation

Dominguez, A. A., Lim, W. A., & Qi, L. S. (2016) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm.2015.2 · Cited by 832

Non–cell autonomous effect of glia on motor neurons in an embryonic stem cell–based ALS model

Di Giorgio, F. P., Carrasco, M. A., Siao, M. C., Maniatis, T., & Eggan, K. (2007) · Nature Neuroscience · doi:10.1038/nn1885 · Cited by 799

Astrocytes from familial and sporadic ALS patients are toxic to motor neurons

Haidet-Phillips, A. M., Hester, M. E., Miranda, C. J., Meyer, K., Braun, L., Frakes, A., Song, S., Likhite, S., Murtha, M. J., Foust, K. D., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.1957 · Cited by 782

Human iPSC-based modeling of late-onset disease via progerin-induced aging

Miller, J. D., Ganat, Y. M., Kishinevsky, S., Bowman, R. L., Liu, B., Tu, E. Y., Mandal, P. K., Vera, E., Shim, J.-W., Kriks, S., et al. (2013) · Cell stem cell · doi:10.1016/j.stem.2013.11.006 · Cited by 777

LRRK2 Mutant iPSC-Derived DA Neurons Demonstrate Increased Susceptibility to Oxidative Stress

Nguyen, H. N., Byers, B., Cord, B. J., Shcheglovitov, A., Byrne, J., Gujar, P., Kee, K., Schüle, B., Dolmetsch, R. E., Langston, J. W., et al. (2011) · Cell stem cell · doi:10.1016/j.stem.2011.01.013 · Cited by 768

Directly Reprogrammed Human Neurons Retain Aging-Associated Transcriptomic Signatures and Reveal Age-Related Nucleocytoplasmic Defects.

Mertens, J., Paquola, A. C. M., Ku, M., Hatch, E., Böhnke, L., Ladjevardi, S., McGrath, S., Campbell, B., Lee, H., Herdy, J. R., et al. (2015) · Cell Stem Cell · doi:10.1016/j.stem.2015.09.001 · Cited by 765

Reprogramming of Mouse and Human Cells to Pluripotency Using Mature MicroRNAs

Miyoshi, N., Ishii, H., Nagano, H., Haraguchi, N., Dewi, D. L., Kano, Y., Nishikawa, S., Tanemura, M., Mimori, K., Tanaka, F., et al. (2011) · Cell stem cell · doi:10.1016/j.stem.2011.05.001 · Cited by 756

Modeling Alzheimer’s Disease with iPSCs Reveals Stress Phenotypes Associated with Intracellular Aβ and Differential Drug Responsiveness

Kondo, T., Asai, M., Tsukita, K., Kutoku, Y., Ohsawa, Y., Sunada, Y., Imamura, K., Egawa, N., Yahata, N., Okita, K., et al. (2013) · Cell Stem Cell · doi:10.1016/j.stem.2013.01.009 · Cited by 741

Tumorigenicity as a clinical hurdle for pluripotent stem cell therapies

Lee, A. S., Tang, C. Z., Rao, M. S., Weissman, I. L., & Wu, J. C. (2013) · Nature Medicine · doi:10.1038/nm.3267 · Cited by 736

A nonviral minicircle vector for deriving human iPS cells

Jia, F., Wilson, K. D., Sun, N., Gupta, D. M., Huang, M., Li, Z., Panetta, N. J., Chen, Z. Y., Robbins, R. C., Kay, M. A., et al. (2010) · Nature Methods · doi:10.1038/nmeth.1426 · Cited by 734

Self-Organization of Polarized Cerebellar Tissue in 3D Culture of Human Pluripotent Stem Cells

Muguruma, K., Nishiyama, A., Kawakami, H., Hashimoto, K., & Sasai, Y. (2015) · Cell Reports · doi:10.1016/j.celrep.2014.12.051 · Cited by 692

Intrinsic Membrane Hyperexcitability of Amyotrophic Lateral Sclerosis Patient-Derived Motor Neurons

Wainger, B. J., Kiskinis, E., Mellin, C., Wiskow, O., Han, S. S. W., Sandoe, J., Perez, N. P., Williams, L. A., Lee, S., Boulting, G. L., et al. (2014) · Cell Reports · doi:10.1016/j.celrep.2014.03.019 · Cited by 676

Characterization of the expression of MHC proteins in human embryonic stem cells

Drukker, M., Katz, G., Urbach, A., Schuldiner, M., Markel, G., Itskovitz‐Eldor, J., Reubinoff, B. E., Mandelboim, O., & Benvenisty, N. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.142298299 · Cited by 671

International SMA Consortium Meeting (26–28 June 1992, Bonn, Germany)

Munsat, T. L., & Davies, K. E. (1992) · Neuromuscular Disorders · doi:10.1016/s0960-8966(06)80015-5 · Cited by 667

A transcription activator-like effector toolbox for genome engineering

Sanjana, N. E., Cong, L., Zhou, Y., Cunniff, M. M., Feng, G., & Zhang, F. (2012) · Nature Protocols · doi:10.1038/nprot.2011.431 · Cited by 643

Epigenetic Memory and Preferential Lineage-Specific Differentiation in Induced Pluripotent Stem Cells Derived from Human Pancreatic Islet Beta Cells

Bar‐Nur, O., Russ, H. A., Efrat, S., & Benvenisty, N. (2011) · Cell stem cell · doi:10.1016/j.stem.2011.06.007 · Cited by 642

Donor cell type can influence the epigenome and differentiation potential of human induced pluripotent stem cells

Kim, K., Zhao, R., Doi, A., Ng, K., Unternaehrer, J., Cahan, P., Huo, H., Loh, Y., Aryee, M. J., Lensch, M. W., et al. (2011) · Nature Biotechnology · doi:10.1038/nbt.2052 · Cited by 613

Incomplete DNA methylation underlies a transcriptional memory of somatic cells in human iPS cells

Ohi, Y., Qin, H., Hong, C., Blouin, L., Polo, J. M., Guo, T., Qi, Z., Downey, S. L., Manos, P. D., Rossi, D. J., et al. (2011) · Nature Cell Biology · doi:10.1038/ncb2239 · Cited by 609

Modeling familial Alzheimer's disease with induced pluripotent stem cells

Yagi, T., Ito, D., Okada, Y., Akamatsu, W., Nihei, Y., Yoshizaki, T., Yamanaka, S., Okano, H., & Suzuki, N. (2011) · Human Molecular Genetics · doi:10.1093/hmg/ddr394 · Cited by 599

Pluripotent stem cells in disease modelling and drug discovery

Avior, Y., Sagi, I., & Benvenisty, N. (2016) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm.2015.27 · Cited by 592

Modeling mouse and human development using organoid cultures

Huch, M., & Koo, B. (2015) · Development · doi:10.1242/dev.118570 · Cited by 578

Disease‐specific phenotypes in dopamine neurons from human iPS‐based models of genetic and sporadic Parkinson's disease

Sánchez‐Danés, A., Richaud‐Patín, Y., Carballo‐Carbajal, I., Jiménez‐Delgado, S., Caig, C., Mora, S., Guglielmo, C. D., Ezquerra, M., Patel, B., Giralt, A., et al. (2012) · EMBO Molecular Medicine · doi:10.1002/emmm.201200215 · Cited by 571

Drug Screening for ALS Using Patient-Specific Induced Pluripotent Stem Cells

Egawa, N., Kitaoka, S., Tsukita, K., Naitoh, M., Takahashi, K., Yamamoto, T., Adachi, F., Kondo, T., Okita, K., Asaka, I., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3004052 · Cited by 566

Differential responses to lithium in hyperexcitable neurons from patients with bipolar disorder

Study, T. P. O. B. D., Mertens, J., Wang, Q.-W., Kim, Y., Yu, D. X., Pham, S., Yang, B., Zheng, Y., Diffenderfer, K. E., Zhang, J., et al. (2015) · Nature · doi:10.1038/nature15526 · Cited by 562

An in vivo model of human small intestine using pluripotent stem cells

Watson, C. L., Mahé, M. M., Múnera, J. O., Howell, J. C., Sundaram, N., Poling, H. M., Schweitzer, J. I., Vallance, J. E., Mayhew, C. N., Sun, Y., et al. (2014) · Nature Medicine · doi:10.1038/nm.3737 · Cited by 561

iPSC-derived neurons from GBA1-associated Parkinson’s disease patients show autophagic defects and impaired calcium homeostasis

Schöndorf, D. C., Aureli, M., McAllister, F. E., Hindley, C. J., Mayer, F., Schmid, B., Sardi, S. P., Valsecchi, M., Hoffmann, S. A., Schwarz, L. K., et al. (2014) · Nature Communications · doi:10.1038/ncomms5028 · Cited by 554

Synaptic dysregulation in a human iPS cell model of mental disorders

Wen, Z., Nguyen, H. N., Guo, Z., Lalli, M. A., Wang, X., Su, Y., Kim, N.-S., Yoon, K.-J., Shin, J., Zhang, C., et al. (2014) · Nature · doi:10.1038/nature13716 · Cited by 549

Generation of functional hippocampal neurons from self-organizing human embryonic stem cell-derived dorsomedial telencephalic tissue

Sakaguchi, H., Kadoshima, T., Soen, M., Narii, N., Ishida, Y., Ohgushi, M., Takahashi, J., Eiraku, M., & Sasai, Y. (2015) · Nature Communications · doi:10.1038/ncomms9896 · Cited by 542

CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs

Mandegar, M. A., Huebsch, N., Frolov, E. B., Shin, E., Truong, A., Olvera, M. P., Chan, A. H., Miyaoka, Y., Holmes, K., Spencer, C. I., et al. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2016.01.022 · Cited by 525

SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

Naryshkin, N. A., Weetall, M., Dakka, A., Narasimhan, J., Zhao, X., Feng, Z., Ling, K. K. Y., Karp, G. M., Qi, H., Woll, M. G., et al. (2014) · Science · doi:10.1126/science.1250127 · Cited by 524

Gene Targeting of a Disease-Related Gene in Human Induced Pluripotent Stem and Embryonic Stem Cells

Zou, J., Maeder, M. L., Mali, P., Pruett‐Miller, S. M., Thibodeau-Beganny, S., Chou, B.-K., Chen, G., Ye, Z., Park, I., Daley, G. Q., et al. (2009) · Cell stem cell · doi:10.1016/j.stem.2009.05.023 · Cited by 513

Genetic Correction of a LRRK2 Mutation in Human iPSCs Links Parkinsonian Neurodegeneration to ERK-Dependent Changes in Gene Expression

Reinhardt, P., Schmid, B., Burbulla, L. F., Schöndorf, D. C., Wagner, L., Glatza, M., Höing, S., Hargus, G., Heck, S. A., Dhingra, A., et al. (2013) · Cell stem cell · doi:10.1016/j.stem.2013.01.008 · Cited by 512

Pharmacological rescue of mitochondrial deficits in iPSC-derived neural cells from patients with familial Parkinson’s disease

Cooper, O., Seo, H., Andrabi, S., Guardia-Laguarta, C., Graziotto, J., Sundberg, M., McLean, J. R., Carrillo-Reid, L., Xie, Z., Osborn, T., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3003985 · Cited by 508

Parkinson-associated risk variant in distal enhancer of α-synuclein modulates target gene expression

Soldner, F., Stelzer, Y., Shivalila, C., Abraham, B. J., Latourelle, J. C., Barrasa, M. I., Goldmann, J. E., Myers, R. H., Young, R. A., & Jaenisch, R. (2016) · Nature · doi:10.1038/nature17939 · Cited by 507

Induced Pluripotent Stem Cells Meet Genome Editing

Hockemeyer, D., & Jaenisch, R. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2016.04.013 · Cited by 499

Parkinson’s disease induced pluripotent stem cells with triplication of the α-synuclein locus

Devine, M. J., Ryten, M., Vodicka, P., Thomson, A. J., Burdon, T., Houlden, H., Cavaleri, F., Nagano, M., Drummond, N. J., Taanman, J.-W., et al. (2011) · Nature Communications · doi:10.1038/ncomms1453 · Cited by 486

Cytosystems dynamics in self-organization of tissue architecture

Sasai, Y. (2013) · Nature · doi:10.1038/nature11859 · Cited by 484

Non-Cell-Autonomous Effect of Human SOD1G37R Astrocytes on Motor Neurons Derived from Human Embryonic Stem Cells

Marchetto, M. C. N., Muotri, A. R., Mu, Y., Smith, A. M., Cezar, G. G., & Gage, F. H. (2008) · Cell Stem Cell · doi:10.1016/j.stem.2008.10.001 · Cited by 475

Low Incidence of Off-Target Mutations in Individual CRISPR-Cas9 and TALEN Targeted Human Stem Cell Clones Detected by Whole-Genome Sequencing

Veres, A., Gosis, B. S., Ding, Q., Collins, R., Ragavendran, A., Brand, H., Erdin, S., Cowan, C. A., Talkowski, M. E., & Musunuru, K. (2014) · Cell Stem Cell · doi:10.1016/j.stem.2014.04.020 · Cited by 474

Mir-302 reprograms human skin cancer cells into a pluripotent ES-cell-like state

Lin, S.-L., Chang, D. C., Chang-Lin, S., Lin, C., Wu, D. T. S., Chen, D. T., & Ying, S. (2008) · RNA · doi:10.1261/rna.1162708 · Cited by 458

Identification and Rescue of α-Synuclein Toxicity in Parkinson Patient–Derived Neurons

Chung, C. Y., Khurana, V., Auluck, P. K., Tardiff, D. F., Mazzulli, J. R., Soldner, F., Baru, V., Lou, Y., Freyzon, Y., Cho, S., et al. (2013) · Science · doi:10.1126/science.1245296 · Cited by 454

Human embryonic stem cell-derived cardiac progenitors for severe heart failure treatment: first clinical case report: Figure 1

Menasché, P., Vanneaux, V., Hagège, A., Bel, A., Cholley, B., Cacciapuoti, I., Parouchev, A., Benhamouda, N., Tachdjian, G., Tosca, L., et al. (2015) · European Heart Journal · doi:10.1093/eurheartj/ehv189 · Cited by 445

Isogenic Human iPSC Parkinson’s Model Shows Nitrosative Stress-Induced Dysfunction in MEF2-PGC1α Transcription

Ryan, S. D., Dolatabadi, N., Chan, S. F., Zhang, X., Akhtar, M. W., Parker, J., Soldner, F., Sunico, C. R., Nagar, S., Talantova, M. V., et al. (2013) · Cell · doi:10.1016/j.cell.2013.11.009 · Cited by 445

Isolation of Human Induced Pluripotent Stem Cell-Derived Dopaminergic Progenitors by Cell Sorting for Successful Transplantation

Doi, D., Samata, B., Katsukawa, M., Kikuchi, T., Morizane, A., Ono, Y., Sekiguchi, K., Nakagawa, M., Parmar, M., & Takahashi, J. B. (2014) · Stem Cell Reports · doi:10.1016/j.stemcr.2014.01.013 · Cited by 441

Neuronal medium that supports basic synaptic functions and activity of human neurons in vitro

Bardy, C., van den Hurk, M., Eames, T., Marchand, C., Hernandez, R. V., Kellogg, M., Gorris, M., Galet, B., Palomares, V., Brown, J., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1504393112 · Cited by 429

iPS cells: a game changer for future medicine

Inoue, H., Nagata, N., Kurokawa, H., & Yamanaka, S. (2014) · The EMBO Journal · doi:10.1002/embj.201387098 · Cited by 414

Next-Generation Regenerative Medicine: Organogenesis from Stem Cells in 3D Culture

Sasai, Y. (2013) · Cell stem cell · doi:10.1016/j.stem.2013.04.009 · Cited by 411

Evolution of strategies to improve preclinical cardiac safety testing

Gintant, G., Sager, P. T., & Stockbridge, N. (2016) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2015.34 · Cited by 407

Identifying genetic risk factors for serious adverse drug reactions: current progress and challenges

Wilke, R. A., Lin, D. W., Roden, D. M., Watkins, P. B., Flockhart, D. A., Zineh, I., Giacomini, K. M., & Krauss, R. M. (2007) · Nature Reviews Drug Discovery · doi:10.1038/nrd2423 · Cited by 400

Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation

Sampaziotis, F., de Brito, M. C., Madrigal, P., Bertero, A., Saeb‐Parsy, K., Soares, F. A. C., Schrumpf, E., Melum, E., Karlsen, T. H., Bradley, J. A., et al. (2015) · Nature Biotechnology · doi:10.1038/nbt.3275 · Cited by 398

Mitochondrial Parkin Recruitment Is Impaired in Neurons Derived from Mutant PINK1 Induced Pluripotent Stem Cells

Seibler, P., Graziotto, J. J., Jeong, H., Šimunović, F., Klein, C., & Krainc, D. (2011) · Journal of Neuroscience · doi:10.1523/jneurosci.4441-10.2011 · Cited by 388

Mitochondrial dysfunction associated with increased oxidative stress and α-synuclein accumulation in PARK2 iPSC-derived neurons and postmortem brain tissue

Imaizumi, Y., Okada, Y., Akamatsu, W., Koike, M., Kuzumaki, N., Hayakawa, H., Nihira, T., Kobayashi, T., Ohyama, M., Sato, S., et al. (2012) · Molecular Brain · doi:10.1186/1756-6606-5-35 · Cited by 385

Pluripotent stem cells progressing to the clinic

Trounson, A. O., & DeWitt, N. D. (2016) · Nature Reviews Molecular Cell Biology · doi:10.1038/nrm.2016.10 · Cited by 380

The familial Alzheimer's disease APPV717I mutation alters APP processing and Tau expression in iPSC-derived neurons

Muratore, C. R., Rice, H. C., Srikanth, P., Callahan, D. G., Shin, T., Benjamin, L. N. P., Walsh, D. M., Selkoe, D. J., & Young-Pearse, T. L. (2014) · Human Molecular Genetics · doi:10.1093/hmg/ddu064 · Cited by 379

Phenotypic differences in hiPSC NPCs derived from patients with schizophrenia

Brennand, K., Savas, J. N., Kim, Y., Tran, N., Simone, A., Hashimoto-Torii, K., Beaumont, K. G., Kim, H. J., Topol, A., Ladran, I., et al. (2015) · Molecular Psychiatry · doi:10.1038/mp.2014.22 · Cited by 369

2D and 3D Stem Cell Models of Primate Cortical Development Identify Species-Specific Differences in Progenitor Behavior Contributing to Brain Size

Otani, T., Marchetto, M. C., Gage, F. H., Simons, B. D., & Livesey, F. J. (2016) · Cell Stem Cell · doi:10.1016/j.stem.2016.03.003 · Cited by 353

Whole-genome sequencing analysis reveals high specificity of CRISPR/Cas9 and TALEN-based genome editing in human iPSCs

Smith, C., Gore, A., Yan, W., Abalde-Atristain, L., Li, Z., He, C., Wang, Y., Brodsky, R. A., Zhang, K., Cheng, L., et al. (2014) · Cell stem cell · doi:10.1016/j.stem.2014.06.011 · Cited by 342

Progressive degeneration of human neural stem cells caused by pathogenic LRRK2

Liu, G.-H., Qu, J., Suzuki, K., Nivet, E., Li, M., Montserrat, N., Yi, F., Xu, X., Ruiz, S., Zhang, W., et al. (2012) · Nature · doi:10.1038/nature11557 · Cited by 339

Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs

Firth, A. L., Menon, T., Parker, G. S., Qualls, S. J., Lewis, B. M., Ke, E., Dargitz, C., Wright, R., Khanna, A., Gage, F. H., et al. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.07.062 · Cited by 337

Human pluripotent stem cell-derived neural constructs for predicting neural toxicity

Schwartz, M. P., Hou, Z., Propson, N. E., Zhang, J., Engstrom, C. J., Costa, V. S., Jiang, P., Nguyen, B. K., Bolin, J. M., Daly, W., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1516645112 · Cited by 325

The $2.6 Billion Pill — Methodologic and Policy Considerations

Avorn, J. (2015) · New England Journal of Medicine · doi:10.1056/nejmp1500848 · Cited by 322

Directed differentiation of cholangiocytes from human pluripotent stem cells

Ogawa, M., Ogawa, S., Bear, C. E., Ahmadi, S., Chin, S., Li, B., Grompe, M., Keller, G. M., Kamath, B. M., & Ghanekar, A. (2015) · Nature Biotechnology · doi:10.1038/nbt.3294 · Cited by 318

Parkinson-causing α-synuclein missense mutations shift native tetramers to monomers as a mechanism for disease initiation

Dettmer, U., Newman, A. J., Soldner, F., Luth, E. S., Kim, N. C., von Saucken, V. E., Sanderson, J. B., Jaenisch, R., Bartels, T., & Selkoe, D. (2015) · Nature Communications · doi:10.1038/ncomms8314 · Cited by 317

A cellular model for sporadic ALS using patient-derived induced pluripotent stem cells

Burkhardt, M. F., Martinez, F. J., Wright, S., Ramos, C., Volfson, D., Mason, M. J., Garnes, J., Dang, V., Lievers, J., Shoukat-Mumtaz, U., et al. (2013) · Molecular and Cellular Neuroscience · doi:10.1016/j.mcn.2013.07.007 · Cited by 316

Selective Elimination of Human Pluripotent Stem Cells by an Oleate Synthesis Inhibitor Discovered in a High-Throughput Screen

Ben‐David, U., Gan, Q.-F., Golan‐Lev, T., Arora, P., Yanuka, O., Oren, Y. S., Leikin-Frenkel, A. I., Graf, M. R., Garippa, R. J., Boehringer, M., et al. (2013) · Cell stem cell · doi:10.1016/j.stem.2012.11.015 · Cited by 312

Human iPSC-derived motoneurons harbouring TARDBP or C9ORF72 ALS mutations are dysfunctional despite maintaining viability

Devlin, A., Burr, K., Borooah, S., Foster, J. D., Cleary, E. M., Geti, I., Vallier, L., Shaw, C. E., Chandran, S., & Miles, G. B. (2015) · Nature Communications · doi:10.1038/ncomms6999 · Cited by 308

Evaluating cell reprogramming, differentiation and conversion technologies in neuroscience

Mertens, J., Marchetto, M. C., Bardy, C., & Gage, F. H. (2016) · Nature reviews. Neuroscience · doi:10.1038/nrn.2016.46 · Cited by 307

Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9

Park, C., Kim, D. H., Sung, J. J., Sung, J. J., Lee, J., Bae, S., Kim, D., Kim, D., & Kim, J. (2015) · Cell stem cell · doi:10.1016/j.stem.2015.07.001 · Cited by 305

Immunosuppressive therapy mitigates immunological rejection of human embryonic stem cell xenografts

Swijnenburg, R., Schrepfer, S., Govaert, J. A., Cao, F., Ransohoff, K., Sheikh, A. Y., Haddad, M., Connolly, A. J., Davis, M. M., Robbins, R. C., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0805802105 · Cited by 298

Translation of Human-Induced Pluripotent Stem Cells

Sayed, N., Liu, C., & Wu, J. C. (2016) · Journal of the American College of Cardiology · doi:10.1016/j.jacc.2016.01.083 · Cited by 290

Single cell transcriptional profiling reveals heterogeneity of human induced pluripotent stem cells

Narsinh, K. H., Sun, N., Sánchez-Freire, V., Lee, A. S., de Almeida, P. E., Hu, S., Jan, T. A., Wilson, K. D., Leong, D. E., Rosenberg, J., et al. (2011) · Journal of Clinical Investigation · doi:10.1172/jci44635 · Cited by 287

In Vitro Differentiation of Embryonic Stem Cells into Hepatocyte‐Like Cells Identified by Cellular Uptake of Indocyanine Green

Yamada, T., Yoshikawa, M., Kanda, S., Kato, Y., Nakajima, Y., Ishizaka, S., & Tsunoda, Y. (2002) · Stem Cells · doi:10.1634/stemcells.20-2-146 · Cited by 287

Current status of pluripotent stem cells: moving the first therapies to the clinic

Kimbrel, E. A., & Lanza, R. (2015) · Nature Reviews Drug Discovery · doi:10.1038/nrd4738 · Cited by 275

Genetic and epigenetic stability of human pluripotent stem cells

Lund, R., Närvä, E., & Lahesmaa, R. (2012) · Nature Reviews Genetics · doi:10.1038/nrg3271 · Cited by 258

Parkin controls dopamine utilization in human midbrain dopaminergic neurons derived from induced pluripotent stem cells

Jiang, H., Ren, Y., Yuen, E. Y., Zhong, P., Ghaedi, M., Hu, Z., Azabdaftari, G., Nakaso, K., Yan, Z., & Feng, J. (2012) · Nature Communications · doi:10.1038/ncomms1669 · Cited by 254

Organoid Models of Human Gastrointestinal Development and Disease

Dedhia, P. H., Bertaux‐Skeirik, N., Zavros, Y. L., & Spence, J. R. (2016) · Gastroenterology · doi:10.1053/j.gastro.2015.12.042 · Cited by 252

RNA-Seq of Human Neurons Derived from iPS Cells Reveals Candidate Long Non-Coding RNAs Involved in Neurogenesis and Neuropsychiatric Disorders

Lin, M., Pedrosa, E., Shah, A., Hrabovsky, A., Maqbool, S. B., Zheng, D., & Lachman, H. M. (2011) · PLoS ONE · doi:10.1371/journal.pone.0023356 · Cited by 247

Inhibition of pluripotent stem cell-derived teratoma formation by small molecules

Lee, M.-O., Moon, S. H., Jeong, H.-C., Yi, J.-Y., Lee, T.-H., Shim, S. H., Rhee, Y.-H., Lee, S.-H., Oh, S.-J., Lee, M.-Y., et al. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1303669110 · Cited by 246

CPAP promotes timely cilium disassembly to maintain neural progenitor pool

Gabriel, E., Wason, A., Ramani, A., Gooi, L. M., Keller, P., Pozniakovsky, A., Poser, I., Noack, F., Telugu, N. S., Calegari, F., et al. (2016) · The EMBO Journal · doi:10.15252/embj.201593679 · Cited by 243

International SMA consortium meeting

Munsat, T. L. (1992) · Medical Entomology and Zoology · Cited by 243

Efficient and Reproducible Myogenic Differentiation from Human iPS Cells: Prospects for Modeling Miyoshi Myopathy In Vitro

Tanaka, A., Woltjen, K., Miyake, K., Hotta, A., Ikeya, M., Yamamoto, T., Nishino, T., Shoji, E., Sehara‐Fujisawa, A., Manabe, Y., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0061540 · Cited by 241

Efficient Generation of Functional Hepatocytes From Human Embryonic Stem Cells and Induced Pluripotent Stem Cells by HNF4α Transduction

Takayama, K., Inamura, M., Kawabata, K., Katayama, K., Higuchi, M., Tashiro, K., Nonaka, A., Sakurai, F., Hayakawa, T., Furue, M. K., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.234 · Cited by 241

Modeling a Genetic Risk for Schizophrenia in iPSCs and Mice Reveals Neural Stem Cell Deficits Associated with Adherens Junctions and Polarity

Yoon, K.-J., Nguyen, H. N., Ursini, G., Zhang, F., Kim, N.-S., Wen, Z., Makri, G., Nauen, D., Shin, J. H., Park, Y., et al. (2014) · Cell Stem Cell · doi:10.1016/j.stem.2014.05.003 · Cited by 240

Comparison of Electrophysiological Data From Human-Induced Pluripotent Stem Cell–Derived Cardiomyocytes to Functional Preclinical Safety Assays

Harris, K. L., Aylott, M. C., Cui, Y., Louttit, J. B., McMahon, N. C., & Sridhar, A. (2013) · Toxicological Sciences · doi:10.1093/toxsci/kft113 · Cited by 237

A New Glucocerebrosidase Chaperone Reduces -Synuclein and Glycolipid Levels in iPSC-Derived Dopaminergic Neurons from Patients with Gaucher Disease and Parkinsonism

Aflaki, E., Borger, D. K., Moaven, N., Stubblefield, B. K., Rogers, S. A., Patnaik, S., Schoenen, F. J., Westbroek, W., Zheng, W., Sullivan, P., et al. (2016) · Journal of Neuroscience · doi:10.1523/jneurosci.0636-16.2016 · Cited by 234

Humanized Mice Reveal Differential Immunogenicity of Cells Derived from Autologous Induced Pluripotent Stem Cells

Zhao, T., Zhang, Z., Westenskow, P. D., Todorova, D., Hu, Z., Lin, T., Rong, Z., Kim, J., He, J., Wang, M., et al. (2015) · Cell stem cell · doi:10.1016/j.stem.2015.07.021 · Cited by 231

Differentiation-defective phenotypes revealed by large-scale analyses of human pluripotent stem cells

Koyanagi‐Aoi, M., Ohnuki, M., Takahashi, K., Okita, K., Noma, H., Sawamura, Y., Teramoto, I., Narita, M., Sato, Y., Ichisaka, T., et al. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1319061110 · Cited by 231

Large-scale screening using familial dysautonomia induced pluripotent stem cells identifies compounds that rescue IKBKAP expression

Lee, G., Ramirez, C. N., Kim, H., Zeltner, N., Liu, B., Radu, C., Bhinder, B., Kim, Y. J., Choi, I. Y., Mukherjee-Clavin, B., et al. (2012) · Nature Biotechnology · doi:10.1038/nbt.2435 · Cited by 230

Human secreted tau increases amyloid-beta production

Bright, J., Hussain, S., Dang, V., Wright, S., Cooper, B., Byun, T., Ramos, C., Singh, A., Parry, G., Stagliano, N., et al. (2015) · Neurobiology of Aging · doi:10.1016/j.neurobiolaging.2014.09.007 · Cited by 230

The Use of Induced Pluripotent Stem Cells in Drug Development

Inoue, H., & Yamanaka, S. (2011) · Clinical Pharmacology & Therapeutics · doi:10.1038/clpt.2011.38 · Cited by 228

iPSC-Derived Dopamine Neurons Reveal Differences between Monozygotic Twins Discordant for Parkinson’s Disease

Woodard, C. M., Campos, B. A., Kuo, S., Nirenberg, M. J., Nestor, M. W., Zimmer, M., Mosharov, E. V., Sulzer, D., Zhou, H., Paull, D. J., et al. (2014) · Cell Reports · doi:10.1016/j.celrep.2014.10.023 · Cited by 226

Efficient Detection and Purification of Cell Populations Using Synthetic MicroRNA Switches

Miki, K., Endo, K., Takahashi, S., Funakoshi, S., Takei, I., Katayama, S., Toyoda, T., Kotaka, M., Takaki, T., Umeda, M., et al. (2015) · Cell stem cell · doi:10.1016/j.stem.2015.04.005 · Cited by 224

Rodent models for Alzheimer’s disease drug discovery

Puzzo, D., Gulisano, W., Palmeri, A., & Arancio, O. (2015) · Expert Opinion on Drug Discovery · doi:10.1517/17460441.2015.1041913 · Cited by 222

Statin treatment rescues FGFR3 skeletal dysplasia phenotypes

Yamashita, A., Morioka, M., Kishi, H., Kimura, T., Yahara, Y., Okada, M., Fujita, K., Sawai, H., Ikegawa, S., & Tsumaki, N. (2014) · Nature · doi:10.1038/nature13775 · Cited by 216

Functional anterior pituitary generated in self-organizing culture of human embryonic stem cells

Ozone, C., Suga, H., Eiraku, M., Kadoshima, T., Yonemura, S., Takata, N., Oiso, Y., Tsuji, T., & Sasai, Y. (2016) · Nature Communications · doi:10.1038/ncomms10351 · Cited by 211

Developing predictive assays: The phenotypic screening “rule of 3”

Vincent, F., Loria, P. M., Pregel, M. J., Stanton, R. V., Kitching, L., Nocka, K. H., Doyonnas, R., Steppan, C. M., Gilbert, A. M., Schroeter, T., et al. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aab1201 · Cited by 204

Programming and Reprogramming Cellular Age in the Era of Induced Pluripotency

Studer, L., Vera, E., & Cornacchia, D. (2015) · Cell stem cell · doi:10.1016/j.stem.2015.05.004 · Cited by 192

Elucidating Molecular Phenotypes Caused by the SORL1 Alzheimer’s Disease Genetic Risk Factor Using Human Induced Pluripotent Stem Cells

Young, J. E., Boulanger-Weill, J., Williams, D. A., Woodruff, G., Buen, F., Revilla, A. C., Herrera, C. M., Israel, M. A., Yuan, S. H., Edland, S. D., et al. (2015) · Cell stem cell · doi:10.1016/j.stem.2015.02.004 · Cited by 189

Short-Term Immunosuppression Promotes Engraftment of Embryonic and Induced Pluripotent Stem Cells

Pearl, J. I., Lee, A. S., Leveson-Gower, D. B., Sun, N., Ghosh, Z., Lan, F., Ransohoff, J. D., Negrin, R. S., Davis, M. M., & Wu, J. C. (2011) · Cell stem cell · doi:10.1016/j.stem.2011.01.012 · Cited by 184

APP Metabolism Regulates Tau Proteostasis in Human Cerebral Cortex Neurons.

Moore, S., Evans, L. D. B., Andersson, T., Portelius, E., Smith, J., Dias, T. B., Saurat, N., McGlade, A., Kirwan, P., Blennow, K., et al. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.03.068 · Cited by 179

The presenilin-1 ΔE9 mutation results in reduced γ-secretase activity, but not total loss of PS1 function, in isogenic human stem cells

Woodruff, G., Young, J. E., Martinez, F. J., Buen, F., Gore, A., Kinaga, J., Li, Z., Yuan, S. H., Zhang, K., & Goldstein, L. S. B. (2013) · Cell Reports · doi:10.1016/j.celrep.2013.10.018 · Cited by 177

Transplanted terminally differentiated induced pluripotent stem cells are accepted by immune mechanisms similar to self-tolerance

de Almeida, P. E., Meyer, E. H., Kooreman, N. G., Diecke, S., Dey, D., Sánchez-Freire, V., Hu, S., Ebert, A., Odegaard, J. I., Mordwinkin, N. M., et al. (2014) · Nature Communications · doi:10.1038/ncomms4903 · Cited by 174

Multi-parameter in vitro toxicity testing of crizotinib, sunitinib, erlotinib, and nilotinib in human cardiomyocytes

Doherty, K. R., Wappel, R. L., Talbert, D. R., Trusk, P. B., Moran, D. M., Kramer, J., Brown, A. M., Shell, S. A., & Bacus, S. S. (2013) · Toxicology and Applied Pharmacology · doi:10.1016/j.taap.2013.04.027 · Cited by 173

Stem Cell-Based Human Blood–Brain Barrier Models for Drug Discovery and Delivery

Aday, S., Cecchelli, R., Hallier-Vanuxeem, D., Dehouck, M. P., & Ferreira, L. (2016) · Trends in Biotechnology · doi:10.1016/j.tibtech.2016.01.001 · Cited by 169

Hurdles to clinical translation of human induced pluripotent stem cells

Neofytou, E. A., O’Brien, C., Couture, L. A., & Wu, J. C. (2015) · Journal of Clinical Investigation · doi:10.1172/jci80575 · Cited by 166

Stem cell derived basal forebrain cholinergic neurons from Alzheimer’s disease patients are more susceptible to cell death

Duan, L., Bhattacharyya, B. J., Belmadani, A. A., Pan, L., Miller, R. J., & Kessler, J. A. (2014) · Molecular Neurodegeneration · doi:10.1186/1750-1326-9-3 · Cited by 165

Transcriptome Profiling of Patient-Specific Human iPSC-Cardiomyocytes Predicts Individual Drug Safety and Efficacy Responses In Vitro

Matsa, E., Burridge, P. W., Yu, K.-H., Ahrens, J. H., Termglinchan, V., Wu, H., Liu, C., Shukla, P., Sayed, N., Churko, J. M., et al. (2016) · Cell stem cell · doi:10.1016/j.stem.2016.07.006 · Cited by 164

Engineered heart tissues and induced pluripotent stem cells: Macro- and microstructures for disease modeling, drug screening, and translational studies

Tzatzalos, E., Abilez, O. J., Shukla, P., & Wu, J. C. (2016) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2015.09.010 · Cited by 162

Toward more predictive genetic mouse models of Alzheimer’s disease

Onos, K. D., Sukoff Rizzo, S. J., Howell, G. R., & Sasner, M. (2016) · Brain Research Bulletin · doi:10.1016/j.brainresbull.2015.12.003 · Cited by 156

Physiological Characterisation of Human iPS-Derived Dopaminergic Neurons

Hartfield, E. M., Yamasaki-Mann, M., Fernandes, H. J. R., Vowles, J., James, W., Cowley, S. A., & Wade‐Martins, R. (2014) · PLoS ONE · doi:10.1371/journal.pone.0087388 · Cited by 152

Acid sphingomyelinase modulates the autophagic process by controlling lysosomal biogenesis in Alzheimer’s disease

Lee, J. K., Jin, H. K., Park, M. H., Kim, B.-R., Lee, P. H., Nakauchi, H., Carter, J., He, X., Schuchman, E. H., & Bae, J. (2014) · The Journal of Experimental Medicine · doi:10.1084/jem.20132451 · Cited by 149

Induced pluripotent stem cell-derived neuronal cells from a sporadic Alzheimer’s disease donor as a model for investigating AD-associated gene regulatory networks

Hossini, A. M., Megges, M., Prigione, A., Lichtner, B., Toliat, M. R., Wruck, W., Schröter, F., Nüernberg, P., Kroll, H., Makrantonaki, E., et al. (2015) · BMC Genomics · doi:10.1186/s12864-015-1262-5 · Cited by 139

Aberrant epigenome in iPSC‐derived dopaminergic neurons from Parkinson's disease patients

Fernández‐Santiago, R., Carballo‐Carbajal, I., Castellano, G., Torrent, R., Richaud, Y., Sánchez‐Danés, A., Vilarrasa‐Blasi, R., Sánchez‐Pla, Á., Mosquera, J. L., Soriano, J., et al. (2015) · EMBO Molecular Medicine · doi:10.15252/emmm.201505439 · Cited by 138

How iPS cells changed the world

Scudellari, M. (2016) · Nature · doi:10.1038/534310a · Cited by 135

Potential Strategies to Address the Major Clinical Barriers Facing Stem Cell Regenerative Therapy for Cardiovascular Disease

Nguyen, P. K. P., Neofytou, E. A., Rhee, J., & Wu, J. C. (2016) · JAMA Cardiology · doi:10.1001/jamacardio.2016.2750 · Cited by 133

Mitochondrial Alterations by PARKIN in Dopaminergic Neurons Using PARK2 Patient-Specific and PARK2 Knockout Isogenic iPSC Lines

Shaltouki, A., Sivapatham, R., Pei, Y., Gerencser, A. A., Momčilović, O., Rao, M. S., & Zeng, X. (2015) · Stem Cell Reports · doi:10.1016/j.stemcr.2015.02.019 · Cited by 131

Predicting drug-induced changes in QT interval and arrhythmias: QT-shortening drugs point to gaps in the ICHS7B Guidelines

Lu, H.-R., Vlaminckx, E., Hermans, A. N., Rohrbacher, J., van Ammel, K., Towart, R., Pugsley, M., & Gallacher, D. (2008) · British Journal of Pharmacology · doi:10.1038/bjp.2008.191 · Cited by 131

Identification of chemicals that mimic transcriptional changes associated with autism, brain aging and neurodegeneration

Pearson, B. L., Simon, J. M., McCoy, E. S., Salazar, G., Fragola, G., & Zylka, M. J. (2016) · Nature Communications · doi:10.1038/ncomms11173 · Cited by 127

Early pathogenesis of Duchenne muscular dystrophy modelled in patient-derived human induced pluripotent stem cells

Shoji, E., Sakurai, H., Nishino, T., Nakahata, T., Heike, T., Awaya, T., FUJII, N. L., Manabe, Y., Matsuo, M., & Sehara‐Fujisawa, A. (2015) · Scientific Reports · doi:10.1038/srep12831 · Cited by 126

From Genomics to Gene Therapy: Induced Pluripotent Stem Cells Meet Genome Editing

Hotta, A., & Yamanaka, S. (2015) · Annual Review of Genetics · doi:10.1146/annurev-genet-112414-054926 · Cited by 124

Dysregulation of miRNA-9 in a Subset of Schizophrenia Patient-Derived Neural Progenitor Cells

Topol, A., Zhu, S., Hartley, B. J., English, J., Hauberg, M. E., Tran, N., Rittenhouse, C. A., Simone, A., Ruderfer, D. M., Johnson, J., et al. (2016) · Cell Reports · doi:10.1016/j.celrep.2016.03.090 · Cited by 122

Induced Pluripotent Stem Cells from Familial Alzheimer's Disease Patients Differentiate into Mature Neurons with Amyloidogenic Properties

Mahairaki, V., Ryu, J., Peters, A., Chang, Q., Li, T., Park, T. S., Burridge, P. W., Talbot, C. C., Asnaghi, L., Martin, L. J., et al. (2014) · Stem Cells and Development · doi:10.1089/scd.2013.0511 · Cited by 120

Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting

Chang, C., Lai, Y.-S., Westin, E. R., Khodadadi‐Jamayran, A., Pawlik, K. M., Lamb, L. S., Goldman, F. D., & Townes, T. M. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.08.013 · Cited by 115

Prevention of β-amyloid induced toxicity in human iPS cell-derived neurons by inhibition of Cyclin-dependent kinases and associated cell cycle events

Xu, X., Lei, Y., Luo, J., Wang, J., Zhang, S., Yang, X., Sun, M., Nuwaysir, E. F., Fan, G.-H., Zhao, J. Y., et al. (2012) · Stem Cell Research · doi:10.1016/j.scr.2012.11.005 · Cited by 115

Parkin mutations reduce the complexity of neuronal processes in iPSC-derived human neurons

Ren, Y., Jiang, H., Hu, Z., Fan, K., Wang, J., Janoschka, S., Wang, X., Ge, S., & Feng, J. (2015) · Stem Cells · doi:10.1002/stem.1854 · Cited by 109

The TLX-miR-219 cascade regulates neural stem cell proliferation in neurodevelopment and schizophrenia iPSC model

Murai, K., Sun, G., Ye, P., Tian, E., Su, Y., Cui, Q., Sun, G., Trinh, D. V., Sun, O., Hong, T., et al. (2016) · Nature Communications · doi:10.1038/ncomms10965 · Cited by 108

ALS disrupts spinal motor neuron maturation and aging pathways within gene co-expression networks

Ho, R., Sances, S., Gowing, G., Mathis, M. W., O’Rourke, J. G., Sahabian, A., Wichterle, H., Baloh, R. H., Sareen, D., & Svendsen, C. N. (2016) · Nature Neuroscience · doi:10.1038/nn.4345 · Cited by 103

Single-Cell Detection of Secreted Aβ and sAPPα from Human IPSC-Derived Neurons and Astrocytes

Liao, M.-C., Muratore, C., Gierahn, T. M., Sullivan, S. E., Srikanth, P., Jager, P. L. D., Love, J. C., & Young‐Pearse, T. L. (2016) · Journal of Neuroscience · doi:10.1523/jneurosci.2735-15.2016 · Cited by 99

Immunosuppression by Embryonic Stem Cells

Koch, C. A., Geraldes, P., & Platt, J. L. (2007) · Stem Cells · doi:10.1634/stemcells.2007-0151 · Cited by 98

Duplication of TBK1 Stimulates Autophagy in iPSC-derived Retinal Cells from a Patient with Normal Tension Glaucoma

Tucker, B. A. (2014) · Journal of Stem Cell Research & Therapy · doi:10.4172/2157-7633.1000161 · Cited by 98

Pluripotent Stem Cells: Immune to the Immune System?

Pearl, J. I., Kean, L. S., Davis, M. M., & Wu, J. C. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3005090 · Cited by 94

Effect of Potent γ-Secretase Modulator in Human Neurons Derived From Multiple Presenilin 1–Induced Pluripotent Stem Cell Mutant Carriers

Liu, Q., Waltz, S., Woodruff, G., Ouyang, J., Israel, M. A., Herrera, C., Sarsoza, F., Tanzi, R. E., Koo, E. H., Ringman, J. M., et al. (2014) · JAMA Neurology · doi:10.1001/jamaneurol.2014.2482 · Cited by 91

From Dish to Bedside: Lessons Learned While Translating Findings from a Stem Cell Model of Disease to a Clinical Trial

McNeish, J. D., Gardner, J. P., Wainger, B. J., Woolf, C. J., & Eggan, K. C. (2015) · Cell stem cell · doi:10.1016/j.stem.2015.06.013 · Cited by 90

Functional Implications of miR-19 in the Migration of Newborn Neurons in the Adult Brain

Han, J., Kim, H. J., Schafer, S. T., Paquola, A. C. M., Clemenson, G. D., Toda, T., Oh, J., Pankonin, A. R., Lee, B. S., Johnston, S. T., et al. (2016) · Neuron · doi:10.1016/j.neuron.2016.05.034 · Cited by 89

High-Throughput Screening Using iPSC-Derived Neuronal Progenitors to Identify Compounds Counteracting Epigenetic Gene Silencing in Fragile X Syndrome

Kaufmann, M., Schuffenhauer, A., Fruh, I., Klein, J., Thiemeyer, A., Rigo, P., Gomez-Mancilla, B., Heidinger-Millot, V., Bouwmeester, T., Schopfer, U., et al. (2015) · SLAS DISCOVERY · doi:10.1177/1087057115588287 · Cited by 87

Single-Cell Gene Expression Profiles Define Self-Renewing, Pluripotent, and Lineage Primed States of Human Pluripotent Stem Cells

Hough, S. R., Thornton, M. E., Mason, E. A., Mar, J. C., Wells, C. A., & Pera, M. F. (2014) · Stem Cell Reports · doi:10.1016/j.stemcr.2014.04.014 · Cited by 84

High-Throughput Screening to Identify Compounds That Increase Fragile X Mental Retardation Protein Expression in Neural Stem Cells Differentiated From Fragile X Syndrome Patient-Derived Induced Pluripotent Stem Cells

Kumari, D., Swaroop, M., Southall, N. T., Huang, W., Zheng, W., & Usdin, K. (2015) · Stem Cells Translational Medicine · doi:10.5966/sctm.2014-0278 · Cited by 83

Proarrhythmia Risk Assessment in Human Induced Pluripotent Stem Cell-Derived Cardiomyocytes Using the Maestro MEA Platform

Qu, Y., & Vargas, H. M. (2015) · Toxicological Sciences · doi:10.1093/toxsci/kfv128 · Cited by 79

Natural products in drug discovery: advances and opportunities

Atanasov, A. G., Zotchev, S. B., Dirsch, V. M., Taskforce, T. I. N. P. S., Orhan, İ. E., Banach, M., Rollinger, J. M., Barreca, D., Weckwerth, W., Bauer, R., et al. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-020-00114-z · Cited by 5,891

COVID-19 and cardiovascular disease: from basic mechanisms to clinical perspectives

Nishiga, M., Wang, D. W., Han, Y., Lewis, D. B., & Wu, J. C. (2020) · Nature Reviews Cardiology · doi:10.1038/s41569-020-0413-9 · Cited by 1,436

Gene therapy clinical trials worldwide to 2012 – an update

Ginn, S. L., Alexander, I. E., Edelstein, M. L., Abedi, M. R., & Wixon, J. (2013) · The Journal of Gene Medicine · doi:10.1002/jgm.2698 · Cited by 1,187

A guide to the organ-on-a-chip

Leung, C. M., de Haan, P., Ronaldson-Bouchard, K., Kim, G.-A., Ko, J., Rho, H. S., Chen, Z., Habibovic, P., Jeon, N. L., Takayama, S., et al. (2022) · Nature Reviews Methods Primers · doi:10.1038/s43586-022-00118-6 · Cited by 1,116

Synthetic alternatives to Matrigel

Aisenbrey, E. A., & Murphy, W. L. (2020) · Nature Reviews Materials · doi:10.1038/s41578-020-0199-8 · Cited by 1,016

Induced pluripotent stem cell technology: venturing into the second decade

Shi, Y., Inoue, H., Takahashi, J. B., & Yamanaka, S. (2020) · Elsevier eBooks · doi:10.1016/b978-0-12-818422-6.00095-2 · Cited by 4

Induced pluripotent stem cell and their applications inDrug Discovery and Precision Personalized Medicine

G, M. V. S. D. S. (2015) · Narayana Medical Journal

Induced pluripotent stem cells for regenerative cardiovascular therapies and biomedical discovery

Nsair, A., & MacLellan, W. R. (2011) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2011.01.013 · Cited by 35

Current Challenges of iPSC-Based Disease Modeling and Therapeutic Implications

Doss, M. X., & Sachinidis, A. (2019) · Cells · doi:10.3390/cells8050403 · Cited by 486

Induced pluripotent stem cells (iPSCs): the emergence of a new champion in stem cell technology-driven biomedical applications

Das, A. K., & Pal, R. (2010) · Journal of Tissue Engineering and Regenerative Medicine · doi:10.1002/term.258 · Cited by 31