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Disease-Specific Induced Pluripotent Stem Cells.
Open accessAbstract
Tissue culture of immortal cell strains from diseased patients is an invaluable resource for medical research but is largely limited to tumor cell lines or transformed derivatives of native tissues. Here we describe the generation of induced pluripotent stem (iPS) cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance; these diseases include adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome. Such disease-specific stem cells offer an unprecedented opportunity to recapitulate both normal and pathologic human tissue formation in vitro, thereby enabling disease investigation and drug development.
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“The first iPSCs reprogrammed from an individual with DS were reported in 2008 (Park et al., 2008) and multiple lines have been created from multiple different individuals since then.”
From Neurodevelopment in Down syndrome: Concordance in humans and models (2022) · cites this paper as Park et al., 2008
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Cites
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“In contrast, introducing a set of transcription factors linked to pluripotency can directly reprogram human somatic cells to produce induced pluripotent stem (iPS) cells, a method that has been achieved by several groups worldwide (Lowry et al., 2008; Park et al., 2008b; Takahashi et al., 2007; Yu et al., 2007).”
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“Following 2 to 3 weeks of culture in hES cell-supporting conditions, compact refractile ES-like colonies emerged among a background of fibroblasts, as previously described (Park et al., 2008a, 2008b).”
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“…from a carrier of Lesch-Nyhan syndrome using five doxycycline-inducible lentiviral vectors (OCT4, SOX2, KLF4, c-MYC, and NANOG), a strategy that has been used to isolate murine iPS cells (Brambrink et al., 2008; Stadtfeld et al., 2008) but previously had not been attempted with human somatic cells.”
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Research works printed but not linked: 1
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“Given the limited availability of heart samples, the use of induced pluripotent stem (iPS) cells to derive heart tissue from fibroblasts or even peripheral blood cells may greatly expand research capabilities.(136,137) • Third, further development of data integration methodology is needed to combine different omics da…”
BackgroundKouroupi, G., Antoniou, N., Prodromidou, K., Taoufik, E., & Matsas, R. (2020) · International Journal of Molecular Sciences · doi:10.3390/ijms21197113 · Cited by 30
Harnessing cellular aging in human stem cell models of amyotrophic lateral sclerosis
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“across a wide spectrum of disease models (Dimos et al., 2008; Hall et al., 2017; Park et al., 2008).”
BackgroundHuman cellular models of medium spiny neuron development and Huntington disease
Golas, M. M. (2018) · Life Sciences · doi:10.1016/j.lfs.2018.07.030 · Cited by 24
Epigenetic-scale comparison of human iPSCs generated by retrovirus, Sendai virus or episomal vectors
Nishino, K., Arai, Y., Takasawa, K., Toyoda, M., Yamazaki-Inoue, M., Sugawara, T., Akutsu, H., Nishimura, K., Ohtaka, M., Nakanishi, M., et al. (2018) · bioRxiv · doi:10.1016/j.reth.2018.08.002 · Cited by 23
“Introduction Human induced pluripotent stem cells (iPSCs) are powerful resources for disease modeling, drug discovery, and regenerative medicine because of their potential for pluripotency, ability to selfrenew indefinitely, avoidance of rejection of their derivatives by the immune system and for ethical issues [1].”
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“iPS cells have ushered in a new era of translational medicine because they can be used to generate patientderived pluripotent stem cell lines that can recapitulate not only disease phenotypes but also the process of disease development [11].”
BackgroundInfluentialCRISPR/Cas9 genome editing in human pluripotent stem cells: Harnessing human genetics in a dish
Gonzãlez, F. (2016) · Developmental Dynamics · doi:10.1002/dvdy.24414 · Cited by 22
“This approach has allowed the generation of a large set of hiPSC lines from patients with a wide range of genetically inherited and sporadic diseases (Park et al., 2008; Wu and Hochedlinger, 2011).”
BackgroundThe emerging role of viral vectors as vehicles for DMD gene editing
Maggio, I., Chen, X., & Gonçalves, M. A. F. V. (2016) · Genome Medicine · doi:10.1186/s13073-016-0316-x · Cited by 27
“These cellular substrates include satellite cells [53, 54] and their committed myoblast progeny [74], induced pluripotent stem cells [75], mesenchymal stromal cells [76, 77], vasculature-associated mesoangioblasts/pericytes [78], and blood-derived CD133 cells [79].”
Neurogenin 2 enhances the generation of patient-specific induced neuronal cells
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Teo, A. K. K., Gupta, M. K., Doria, A., & Kulkarni, R. (2015) · Molecular Metabolism · doi:10.1016/j.molmet.2015.06.006 · Cited by 26
Wang, L., & Leung, P. (2013) · Molecular and Cellular Endocrinology · doi:10.1016/j.mce.2013.08.008 · Cited by 23
“The reprogrammed cells resemble native b-cells in appearance and gene expression, and are also able to partially restore blood sugar regulation in mice whose own b-cells have been chemically destroyed (Dimos et al., 2008; Maehr et al., 2009; Park et al., 2008; Zhou et al., 2008).”
BackgroundToward pluripotency by reprogramming: mechanisms and application
Wang, T., Warren, S. T., & Jin, P. (2013) · Protein & Cell · doi:10.1007/s13238-013-3074-1 · Cited by 26
“…and subsequently differentiating them into the desired cell types will give us new insights into the pathogenesis of a broad spectrum of diseases (Park et al., 2008; Chambers et al., 2009; Merkle and Eggan, 2013). iPS cell lines from patients with different syndromes have been successfully…”
BackgroundFunctions of Huntingtin in Germ Layer Specification and Organogenesis
Nguyen, G. D., Molero, A. E., Gokhan, S., & Mehler, M. (2013) · PLoS ONE · doi:10.1371/journal.pone.0072698 · Cited by 23
Pasquale, E. D., Song, B., & Condorelli, G. (2013) · Journal of Visualized Experiments · doi:10.3791/50429 · Cited by 15
Bioengineered stem cells in neural development and neurodegeneration research
Yuan, S. H., & Shaner, M. A. (2013) · Ageing Research Reviews · doi:10.1016/j.arr.2013.04.002 · Cited by 24
The Specification of Telencephalic Glutamatergic Neurons from Human Pluripotent Stem Cells
Boisvert, E. M., Denton, K. R., Lei, L., & Li, X. (2013) · Journal of Visualized Experiments · doi:10.3791/50321 · Cited by 37
Treatment options for diabetes: Potential role of stem cells
Stanekzai, J., Isenović, E. R., & Mousa, S. A. (2012) · Diabetes Research and Clinical Practice · doi:10.1016/j.diabres.2012.09.010 · Cited by 24
“Interestingly, iPS cells can be derived from somatic cells, which presents the possibility of generating patient-specific pluripotent cells [55].”
BackgroundInfluentialExcision of a viral reprogramming cassette by delivery of synthetic Cre mRNA
Loh, Y., Yang, J., Angeles, A. D. L., Guo, C.-G., Cherry, A. B. C., Rossi, D. J., Park, I.-H., & Daley, G. (2012) · Current protocols in stem cell biology · doi:10.1002/9780470151808.sc04a05s21 · Cited by 23
Self-citationSong, M., Paul, S., Lim, H., Dayem, A. A., & Cho, S. (2012) · Archives of Pharmacal Research · doi:10.1007/s12272-012-0205-9 · Cited by 23
Understanding the Molecular Circuitry of Cell Lineage Specification in the Early Mouse Embryo
Bergsmedh, A., Donohoe, M. E., Hughes, R., & Hadjantonakis, A. (2011) · Genes · doi:10.3390/genes2030420 · Cited by 18
“) in order to restore normal function [142].”
BackgroundUsing human pluripotent stem cells to untangle neurodegenerative disease mechanisms
Malgrange, B., Borgs, L., Grobarczyk, B., Purnelle, A., Ernst, P., Moonen, G., & Nguyen, L. (2010) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-010-0557-6 · Cited by 16
“Consistently, disease-specific hiPS cell lines have recently been engineered to untangle the pathological events of neurodegenerative disorders, including Parkinson’s or Huntington’s diseases [165, 166].”
Gene therapy for β-thalassaemia: the continuing challenge
Yannaki, E., Emery, D. W., & Stamatoyannopoulos, G. (2010) · Expert Reviews in Molecular Medicine · doi:10.1017/s1462399410001626 · Cited by 21
“135 Park, I. et al. (2008) Disease-specific induced pluripotent stem cells.”
Variation in Hematopoietic Potential of Induced Pluripotent Stem Cell Lines
Kulkeaw, K., Horio, Y., Mizuochi, C., Ogawa, M., & Sugiyama, D. (2010) · Stem Cell Reviews and Reports · doi:10.1007/s12015-010-9150-5 · Cited by 23
“Moreover, disease-specific iPS cells may be useful to analyze mechanisms underlying hematological disease [24].”
BackgroundSermon, K., Simón, C., Braude, P., Viville, S., Borstlap, J., & Veiga, A. (2009) · Human Reproduction · doi:10.1093/humrep/dep062 · Cited by 23
“Nevertheless, researchers have already set up a library with 20 different iPSC lines that carry different diseases such as Huntington’s disease (Park et al., 2008, www.the-scientist.com of 7 August 2008), commenting that this is the first time that these cells are available in vitro.”
BackgroundJonsson, M. K. B., van Veen, T. V., Goumans, M., Vos, M., Duker, G., & Sartipy, P. (2009) · Expert Opinion on Drug Discovery · doi:10.1517/17460440902794912 · Cited by 23
“Generation of iPS cells from patients with 10 different genetic diseases, including Parkinson disease and type 1 diabetes mellitus, has already been performed indicating a rapid progress in this field [97].”
BackgroundPassage number affects differentiation of sensory neurons from human induced pluripotent stem cells
Cantor, E. L., Shen, F., Jiang, G., Tan, Z., Cunningham, G. M., Wu, X., Philips, S., & Schneider, B. (2022) · Scientific Reports · doi:10.1038/s41598-022-19018-6 · Cited by 22
Induced Pluripotent Stem Cell-Based Drug Screening by Use of Artificial Intelligence
Kusumoto, D., Yuasa, S., & Fukuda, K. (2022) · Pharmaceuticals · doi:10.3390/ph15050562 · Cited by 37
“great interest in using disease-specific iPSCs in the development of novel treatments for genetically intractable diseases for which no treatment exists [12,13].”
BackgroundEfficient induction and sustenance of pluripotent stem cells from bovine somatic cells
Pillai, V. V., Koganti, P. P., Kei, T. G., Gurung, S., Butler, W. R., & Selvaraj, V. (2021) · Biology Open · doi:10.1242/bio.058756 · Cited by 22
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“Following the demonstration that human cells were indeed reprogrammable, the cells of human patients were reprogrammed yielding the starting material for a new frontier of in vitro disease modeling (Park et al., 2008).”
BackgroundAddressing Manufacturing Challenges for Commercialization of iPSC-Based Therapies
Dashtban, M., Panchalingam, K. M., Shafa, M., & Baghbaderani, B. A. (2020) · Methods in molecular biology · doi:10.1007/7651_2020_288 · Cited by 22
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“[30], expecting to detect stronger effects of mHTT on bioenergetics in this cell line.”
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Multiple system atrophy: experimental models and reality
Overk, C., Rockenstein, E. M., Valera, E., Stefanova, N., Wenning, G. K., & Masliah, E. (2017) · Acta Neuropathologica · doi:10.1007/s00401-017-1772-0 · Cited by 23
“Other in vitro models have employed glioblastoma/ astrocytoma (U373) cells [128] or mixed rat glial cultures [125] transfected with full-length or C-terminally truncated (1-111) α-syn; however, they experience some drawbacks [96].”
BackgroundYu, S. B., Baek, J., Choi, M., Oh, Y., Lee, H., Yu, S. J., Lee, E., Sohn, J.-W., Im, S., & Jon, S. (2016) · ACS Nano · doi:10.1021/acsnano.6b03527 · Cited by 22
Massa, M., Gisevius, B., Hirschberg, S., Hinz, L., Schmidt, M., Gold, R., Prochnow, N., & Haghikia, A. (2016) · PLoS ONE · doi:10.1371/journal.pone.0155274 · Cited by 22
“Because there is evidence of both genomic stability [15,22,23] and epigenetic persistence [24] through the procedure of iPSC conversion and differentiation, the cell- and patient-specific in vitro paradigmmay not only contain relevant genetic material, but also epigenetic information necessary for the investigation of…”
BackgroundMedhekar, S. K., Shende, V. S., & Chincholkar, A. B. (2016) · International Journal of Stem Cells · doi:10.15283/ijsc.2016.9.1.21 · Cited by 24
“Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome (65).”
BackgroundLiu, L., Huang, J., Han, C., Zhang, G., Xu, X., Shen, Y., Li, J., Jiang, H., Lin, Z., Xiong, N., et al. (2015) · Molecular Neurobiology · doi:10.1007/s12035-015-9601-8 · Cited by 23
“HD patient fibroblast 72 CAG repeats No apparent difference from control iPSCs [57]”
BackgroundReprogramming cancer cells: A novel approach for cancer therapy or a tool for disease-modeling?
Yılmazer, A., de Lázaro, I., & Taheri, H. (2015) · Cancer Letters · doi:10.1016/j.canlet.2015.06.027 · Cited by 25
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“The unique attributes of iPS are the generation of patientand disease-specific cells for early drug discovery and safety assessment [85-89].”
BackgroundCurrent status of induced pluripotent stem cells in cardiac tissue regeneration and engineering
Liu, Z., Zhou, J., Wang, H., Zhao, M., & Wang, C. (2013) · Regenerative Medicine Research · doi:10.1186/2050-490x-1-6 · Cited by 20
Abdelli, L. S., Merino, H., Rocher, C., & Singla, D. K. (2012) · Canadian Journal of Physiology and Pharmacology · doi:10.1139/y11-130 · Cited by 13
“Additionally, these cells offer great promise in terms of regenerative medicine as they offer the possibility to produce patient specific iPS cells, which would be very useful for generating disease models and conducting drug screenings for a variety of diseases (Park et al. 2008; Ebert et al. 2009; Carvajal-Vergara e…”
BackgroundInduced human pluripotent stem cells and advanced therapies
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Stem cell-derived neurotrophic support for the neuromuscular junction in spinal muscular atrophy
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“Recently, patient-specific induced pluripotent stem (iPS) cells have been generated from human adult somatic tissues [57-62] as well as from a variety of diseases including amyotrophic lateral sclerosis, muscular dystrophy and Huntington’s disease [57,63,64].”
BackgroundPhillips, B. W., & Crook, J. M. (2010) · BioDrugs · doi:10.2165/11532270-000000000-00000 · Cited by 22
“Examples include trisomy 21, Huntington disease, amyotrophic lateral sclerosis, Gaucher disease, Duchenne muscular dystrophy, and type 1 diabetes, among others,([33,34,81,82]) with POC for drug screening applications provided using spinal muscular atrophy (SMA)([83]) and familial dysautonomia([84]) cell lines.”
BackgroundDifferentiation of Human Induced Pluripotent Stem Cells to Chondrocytes.
Guzzo, R. M., & Drissi, H. (2015) · Methods in molecular biology · doi:10.1007/978-1-4939-2938-2_6 · Cited by 22
Isolation, expansion, and characterization of human islet-derived progenitor cells.
Joglekar, M., & Hardikar, A. (2012) · Methods in molecular biology · doi:10.1007/978-1-61779-815-3_21 · Cited by 22
Baharvand, H., Totonchi, M., Taei, A., Seifinejad, A., Aghdami, N., & Salekdeh, G. (2010) · Methods in molecular biology · doi:10.1007/978-1-60761-369-5_23 · Cited by 22
Ackermann, M., Saleh, F., Abdin, S. M., Hashtchin, A. R., Gensch, I., Golgath, J., Oliveira, M. C., Nguyen, A. H. H., Gaedcke, S., Fenske, A., et al. (2024) · Stem Cell Research & Therapy · doi:10.1186/s13287-024-03785-2 · Cited by 24
“This underscores the need for more physiological test systems, such as hiPSC-derived models to accurately recapitulate the disease and ultimately develop successful drug candidates or therapies [10–12].”
Latoszek, E., & Czeredys, M. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.657337 · Cited by 24
Harnessing the Potential of Stem Cells for Disease Modeling: Progress and Promises
Argentati, C., Tortorella, I., Bazzucchi, M., Morena, F., & Martino, S. (2020) · Journal of Personalized Medicine · doi:10.3390/jpm10010008 · Cited by 24
“Lesch-Nyhan syndrome Patient-derived fibroblasts [53]”
InfluentialRecapitulating and Correcting Marfan Syndrome in a Cellular Model
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“Patient-derived induced pluripotent stem cells (iPSCs) have offered unprecedented opportunities to model [14-16] and genetically treat diseases [17-20].”
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“Human iPS cells also provide a unique platform for studying genetic diseases in vitro (Park et al., 2008).”
BackgroundCell replacement therapy for central nervous system diseases
McKinnon, R. D., & Tso, D. (2015) · Neural Regeneration Research · doi:10.4103/1673-5374.165209 · Cited by 23
“The process appears to work on any cell type (Park et al., 2008b; Soldner et al., 2009; Yu et al., 2009) including readily accessible cells such as adipose-derived mesenchymal stromal cells (MSCs), and iPS lines have now been established from many sources (Park et al., 2008a; Soldner et al., 2009).”
BackgroundNanomedicine-Based Neuroprotective Strategies in Patient Specific-iPSC and Personalized Medicine
Jang, S.-F., Liu, W., Song, W.-S., Chiang, K.-L., Ma, H.-I., Kao, C.-L., & Chen, M.-T. (2014) · International Journal of Molecular Sciences · doi:10.3390/ijms15033904 · Cited by 17
“Recently human iPSCs were successfully established into cell lines from a HD patient with a 72-repeat CAG tract [76].”
BackgroundGao, A., Peng, Y., Deng, Y., & Qing, H. (2012) · Neuroscience · doi:10.1016/j.neuroscience.2012.09.076 · Cited by 20
“To produce both normal and pathological human tissues in vitro for the purpose of systematic disease pathophysiology investigation, Daley’s group (Park et al., 2008) generated a large number of iPSCs from patients with a variety of genetic diseases such as Parkinson’s Disease (PD), Huntington’s Disease (HD), juvenile-…”
BackgroundLiu, J., Sümer, H., Leung, J., Upton, K. R., Dottori, M., Pébay, A., & Verma, P. J. (2010) · Cell Transplantation · doi:10.3727/096368910x514305 · Cited by 18
Stem cells: a review and implications for urology.
Yu, R. N., & Estrada, C. R. (2010) · Urology · doi:10.1016/j.urology.2009.03.058 · Cited by 21
Human stem cell‐based models for studying host‐pathogen interactions
Pellegrino, E., & Gutierrez, M. G. (2021) · Cellular Microbiology · doi:10.1111/cmi.13335 · Cited by 23
“Gene editing approaches allow the generation of isogenic pairs of cells harbouring or not a specific diseasecausing mutation (I. H. Park et al., 2008).”
BackgroundMethodGeneration of neural cells using iPSCs from sleep bruxism patients with 5-HT2A polymorphism.
Hoashi, Y., Okamoto, S., Abe, Y., Matsumoto, T., Tanaka, J., Yoshida, Y., Imaizumi, K., Mishima, K., Akamatsu, W., Okano, H., et al. (2017) · Journal of Prosthodontic Research · doi:10.1016/j.jpor.2016.11.003 · Cited by 20
Induced pluripotent stem cells as a cellular model for studying Down Syndrome
Brigida, A., & Siniscalco, D. (2016) · Journal of Stem Cells and Regenerative Medicine · doi:10.46582/jsrm.1202009 · Cited by 20
Burbulla, L. F., Beaumont, K. G., Mrksich, M., & Krainc, D. (2016) · Advanced Healthcare Materials · doi:10.1002/adhm.201500900 · Cited by 22
Three-dimensional imaging of human stem cells using soft X-ray tomography
Niclis, J. C., Murphy, S. V., Parkinson, D. Y., Zedan, A., Sathananthan, A. H., Cram, D. S., & Heraud, P. (2015) · Journal of The Royal Society Interface · doi:10.1098/rsif.2015.0252 · Cited by 21
“As such, this class of cells is an invaluable research tool for regenerative medicine, which aims to replace or repair damaged tissue [1–3], as well as enabling disease modelling, whereby stem cells with specific genetic or functional defects provide an opportunity to interrogate human pathologies in vitro [4–7].”
BackgroundJacquet, L., Neueder, A., Foldes, G., Karagiannis, P., Hobbs, C., Jolinon, N., Mioulane, M., Sakai, T., Harding, S., & Ilic, D. (2015) · PLoS ONE · doi:10.1371/journal.pone.0126860 · Cited by 20
Khalifian, S., Sarhane, K. A., Tammia, M., Ibrahim, Z., Mao, H., Cooney, D. S., Shores, J. T., Lee, W. P. A., & Brandacher, G. (2014) · Archivum Immunologiae et Therapiae Experimentalis · doi:10.1007/s00005-014-0323-9 · Cited by 24
“…Stem Cells Recent advances in induced pluripotent stem (iPS) cell research and technology have enabled the generation of patient-specific cell lines that may be utilized as a therapeutic modality in various disorders (Dimos et al. 2008; Ebert et al. 2009; Lee et al. 2009; Park et al. 2008).”
BackgroundXie, Y., & Zhang, R. X. (2014) · Neurological Sciences · doi:10.1007/s10072-014-1989-9 · Cited by 24
“The first established human HD-iPSC model from HD patients carrying 72 CAG repeats was reported in 2008 [33].”
BackgroundSolving the puzzle of Parkinson’s disease using induced pluripotent stem cells
Zhao, P., Luo, Z., Tian, W., Yang, J., Ibañez, D. P., Huang, Z., Tortorella, M. D., Esteban, M. A., & Fan, W. (2014) · Experimental Biology and Medicine · doi:10.1177/1535370214538588 · Cited by 16
“PD in a dish using iPSCs Park et al.(18) were the first to report the generation of PD iPSCs (from a sporadic patient), but this early work focused on technical aspects of the reprogramming method, and the disease phenotype was not explored.”
MethodSatoh, D., Maeda, T., Ito, T., Nakajima, Y., Ohte, M., Ukai, A., Nakamura, K., Enosawa, S., Toyota, M., Miyagawa, Y., et al. (2013) · Genes to Cells · doi:10.1111/gtc.12101 · Cited by 21
“Pluripotent cells are by definition capable of differentiating into cell types derived from each of the three embryonic germ layers (Dimos et al. 2008; Park et al. 2008).”
BackgroundDevelopment of nuclease-mediated site-specific genome modification
Wirt, S. E., & Porteus, M. H. (2012) · Current Opinion in Immunology · doi:10.1016/j.coi.2012.08.005 · Cited by 21
Huang, H., Chuang, C.-Y., & Kuo, H. (2012) · Stem Cell Research & Therapy · doi:10.1186/scrt125 · Cited by 21
Programming and reprogramming neuronal subtypes in the central nervous system
Rouaux, C., Bhai, S. F., & Arlotta, P. (2012) · Developmental Neurobiology · doi:10.1002/dneu.22018 · Cited by 20
“These include lines from patients with Huntington’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, spinal muscular atrophy, schizophrenia and Down syndrome (Dimos et al., 2008; Park et al., 2008; Ebert et al., 2009; Soldner et al., 2009; Brennand et al., 2011; Chiang et al., 2011; Marchetto et al., 2011;…”
BackgroundInfluentialTracing the Conversion Process from Primordial Germ Cells to Pluripotent Stem Cells in Mice1
Nagamatsu, G., Kosaka, T., Saito, S., Takubo, K., Akiyama, H., Sudo, T., Horimoto, K., Oya, M., & Suda, T. (2012) · Biology of Reproduction · doi:10.1095/biolreprod.111.096792 · Cited by 18
“Several recent studies also report improved induction methods, including integration-free induction of reprogramming factors [3–9].”
BackgroundConcise Review: The Magic Act of Generating Induced Pluripotent Stem Cells: Many Rabbits in the Hat
Mostoslavsky, G. (2011) · Stem Cells · doi:10.1002/stem.742 · Cited by 20
Induced Pluripotent Stem Cells—A New Foundation in Medicine
Huang, G. T. (2010) · Journal of Experimental & Clinical Medicine · doi:10.1016/s1878-3317(10)60033-2 · Cited by 20
Induced pluripotent stem cells – alchemist's tale or clinical reality?
Rashid, S. T., & Vallier, L. (2010) · Expert Reviews in Molecular Medicine · doi:10.1017/s1462399410001596 · Cited by 17
“22 Park, I.H. (2008) Disease-specific induced pluripotent stem cells.”
Generation of functionally mature neutrophils from induced pluripotent stem cells.
Sweeney, C. L., Merling, R., Choi, U., Priel, D. L. L., Kuhns, D., Wang, H., & Malech, H. (2014) · Methods in molecular biology · doi:10.1007/978-1-62703-845-4_12 · Cited by 20
Activin/nodal signaling and pluripotency.
Chng, Z., Vallier, L., & Pedersen, R. (2011) · Vitamins and hormones · doi:10.1016/b978-0-12-385961-7.00003-2 · Cited by 20
Animal modeling an oligodendrogliopathy – multiple system atrophy
Bleasel, J. M., Halliday, G. M., & Kim, W. S. (2016) · Acta Neuropathologica Communications · doi:10.1186/s40478-016-0279-6 · Cited by 19
Cell reprogramming: methods, mechanisms and applications
Zhu, F., & Nie, G. (2025) · Cell Regeneration · doi:10.1186/s13619-025-00229-x · Cited by 26
“The iPSC technology used to mimic human diseases is characterized by the fact that iPSCs can be reprogrammed from somatic cells of patients with specific diseases and carrying pathogenic mutations or genetic risk factors (Park et al. 2008a).”
InfluentialThe iNs and Outs of Direct Reprogramming to Induced Neurons
Carter, J., Halmai, J. A. N. M., & Fink, K. D. (2020) · Frontiers in Genome Editing · doi:10.3389/fgeed.2020.00007 · Cited by 21
“Differentiating iPSCs to the neuronal-cell fate uncovered diverse neuronal networks associated with reprogramming and has benefited disease modeling efforts for neurodegenerative disorders (Dimos et al., 2008; Park et al., 2008; Chambers et al., 2009; Soldner et al., 2009; Marchetto et al., 2010; Urbach et al., 2010;…”
BackgroundCavalli, E., Battaglia, G., Basile, M. S., Bruno, V., Petralia, M. C., Lombardo, S. D., Pennisi, M., Kalfin, R. E., Tancheva, L. P., Fagone, P., et al. (2020) · Brain Sciences · doi:10.3390/brainsci10030166 · Cited by 26
“The generation of iPSCs derived neuronal cells from patients with AD represent a unique opportunity to create a relevant in vitro model for mechanistic studies and preclinical drug discovery, and have been widely exploited in AD [7–12], as well as in other diseases such as amyotrophic lateral sclerosis [13], Parkinson…”
BackgroundGene delivery methods and genome editing of human pluripotent stem cells
Czerwińska, P., Mazurek, S., Kołodziejczak, I., & Wiznerowicz, M. (2019) · Reports of Practical Oncology & Radiotherapy · doi:10.1016/j.rpor.2019.01.007 · Cited by 20
Emerging technologies for prediction of drug candidate efficacy in the preclinical pipeline
Menshykau, D. (2017) · Drug Discovery Today · doi:10.1016/j.drudis.2017.04.019 · Cited by 27
Genome editing in pluripotent stem cells: research and therapeutic applications
Deleidi, M., & Yu, C. (2016) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2016.02.113 · Cited by 20
Wang, L.-H., Li, X., Huang, W.-H., Zhou, T., Wang, H., Lin, A.-P., Hutchins, A., Su, Z., Chen, Q.-Y., Pei, D., et al. (2016) · Scientific Reports · doi:10.1038/srep22484 · Cited by 19
Lopes, C., & Rego, A. C. (2016) · Molecular Neurobiology · doi:10.1007/s12035-016-9714-8 · Cited by 18
Genome Editing Gene Therapy for Duchenne Muscular Dystrophy
Hotta, A. (2015) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-150116 · Cited by 23
“So far, hundreds of patient-derived iPS cell lines have been established, including DMD patient-derived iPS cells, which were first reported in 2008 [84].”
BackgroundDirect Reprogramming into Desired Cell Types by Defined Factors
Ieda, M. (2013) · The Keio Journal of Medicine · doi:10.2302/kjm.2012-0017-re · Cited by 20
Expanded complexity of unstable repeat diseases
Polak, U., McIvor, E., Dent, S., Wells, R. D., & Напиерала, М. (2012) · BioFactors · doi:10.1002/biof.1060 · Cited by 19
Establishment of automated culture system for murine induced pluripotent stem cells
Koike, H., Kubota, K., Sekine, K., Takebe, T., Ouchi, R., Zheng, Y.-W., Ueno, Y., Tanigawa, N., & Taniguchi, H. (2012) · BMC Biotechnology · doi:10.1186/1472-6750-12-81 · Cited by 19
Using Human-Induced Pluripotent Stem Cells to Model Monogenic Metabolic Disorders of the Liver
Ordonez, M. P., & Goldstein, L. (2012) · Seminars in liver disease (Print) · doi:10.1055/s-0032-1329898 · Cited by 19
How can human pluripotent stem cells help decipher and cure Huntington's disease?
Perrier, A., & Peschanski, M. (2012) · Cell Stem Cell · doi:10.1016/j.stem.2012.07.015 · Cited by 19
Benevento, M., & Muñoz, J. (2012) · Expert Review of Proteomics · doi:10.1586/epr.12.30 · Cited by 14
The Janus face of pluripotent stem cells – Connection between pluripotency and tumourigenicity
Wobus, A. M. (2010) · BioEssays · doi:10.1002/bies.201000065 · Cited by 15
Illuminating the Potential of Pluripotent Stem Cells
Rosenzweig, A. (2010) · New England Journal of Medicine · doi:10.1056/nejme1007902 · Cited by 18
Environmental epigenetic modifications and reprogramming-recalcitrant genes
Sakurada, K. (2010) · Stem Cell Research · doi:10.1016/j.scr.2010.01.001 · Cited by 23
Induced Pluripotent Stem Cells: A New Approach for Physiological Research
Pfannkuche, K., Hannes, T., Khalil, M., Noghabi, M. S., Morshedi, A., Hescheler, J., & Dröge, P. (2010) · Cellular Physiology and Biochemistry · doi:10.1159/000320514 · Cited by 21
Metadata
- Authors
- Park, I.-H., Arora, N., Huo, H., Maherali, N., Ahfeldt, T., Shimamura, A., Lensch, M. W., Cowan, C., Hochedlinger, K., & Daley, G. Q.
- Affiliations
- Boston Children's Hospital (US)
- Harvard University (US)
- University of Washington (US)
- Brigham and Women's Hospital (US)
- Corresponding author
- Daley, George Q.
- ORCID
- Venue
- Cell
- Publisher
- Elsevier BV
- Published
- 2008-09
- Volume
- 134
- Issue
- 5
- Pages
- 877-886
- ISSN
- 0092-8674 (ISSN-L)
- PMC
- PMC2633781
- OpenAlex
- W2157645703
- Cited by
- 2,196
- References
- 34
- Citations by year
- 2026: 20 · 2025: 32 · 2024: 38 · 2023: 51 · 2022: 62 · 2021: 75 · 2020: 89 · 2019: 73 · 2018: 71 · 2017: 108 · 2016: 125 · 2015: 144 · 2014: 139 · 2013: 204 · 2012: 272
- FWCI
- 95.00
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Cell (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- Howard Hughes Medical Institute
- National Institutes of Health (DP1OD000256)
- National Heart, Lung, and Blood Institute (R01HL079582)
- National Institute of Diabetes and Digestive and Kidney Diseases (R01DK059279)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Pluripotent Stem Cells Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Cell Biology › Mesenchymal stem cell research
- Keywords
- induced pluripotent stem cells, genetic disorders, Mendelian inheritance, multifactorial inheritance, ADA-SCID, Duchenne muscular dystrophy, Becker muscular dystrophy, Parkinson's disease, Huntington's disease, Down syndrome, Lesch-Nyhan syndrome, in vitro disease model, drug development
- MeSH terms
- Bone Marrow Cells, Cell Line, Fibroblasts, Humans, Karyotyping, Mutation, Genetic Diseases, Inborn, Pluripotent Stem Cells, Mesenchymal Stem Cells
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