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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
Induction of pluripotent stem cells from adult human fibroblasts by defined factors.
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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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“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).”
Reprogramming of human somatic cells to pluripotency with defined factors
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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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“…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.”
MethodInfluentialSelf-citationNon–cell autonomous effect of glia on motor neurons in an embryonic stem cell–based ALS model
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- Hirschhorn, 1993, Two new mutations at the adenosine deaminase (ADA) locus (Q254X and del nt1050–54) unusual for not being missense mutations, Hum. Mutat., 2, 320, 10.1002/humu.1380020415 · DOI
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Items 601–700 of 1,010
Tumors Originating from Induced Pluripotent Stem Cells and Methods for Their Prevention
Duinsbergen, D., Salvatori, D., Eriksson, M., & Mikkers, H. (2009) · Annals of the New York Academy of Sciences · doi:10.1111/j.1749-6632.2009.04563.x · Cited by 45
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“Although the first generation of iPSCs could not be used for human therapies due to the viral methods used for deriving them, they opened up the possibility of studying patient-specific hereditary diseases using their own cells (281-284).”
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Generation of male differentiated germ cells from various types of stem cells
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“Due to human iPS cells can be generated from patients’ somatic cells, a large number of patient-specific human iPS cell lines have been established and might be used for reproductive medicine (Yamanaka 2007, Dimos et al. 2008, Park et al. 2008, Saha & Jaenisch 2009).”
BackgroundInduced neural stem cells: Methods of reprogramming and potential therapeutic applications
Ruggieri, M., Riboldi, G. M., Brajkovic, S., Bucchia, M., Bresolin, N., Comi, G. P., & Corti, S. P. (2013) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2013.11.001 · Cited by 44
Stem cell biology and drug discovery
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Induced pluripotent stem cells: A new technology to study human diseases
Vitale, A. M., Wolvetang, E. J., & Mackay‐Sim, A. (2011) · The International Journal of Biochemistry & Cell Biology · doi:10.1016/j.biocel.2011.03.013 · Cited by 39
“Some of the limitations that animal models have are due to their genomic differences, in terms of genetic background and number of chromosomes (Park et al., 2008; Saha and Jaenisch, 2009).”
BackgroundNew perspectives in stem cell research: beyond embryonic stem cells
Leeb, C., Jurga, M., McGuckin, C., Forraz, N., Thallinger, C., Moriggl, R., & Kenner, L. (2011) · Cell Proliferation · doi:10.1111/j.1365-2184.2010.00725.x · Cited by 44
Winkler, T., Cantilena, A. R., Métais, J., Xu, X., Nguyen, A., Borate, B. R., Antosiewicz‐Bourget, J., Wolfsberg, T. G., Thomson, J. A., & Dunbar, C. E. (2010) · Stem Cells · doi:10.1002/stem.322 · Cited by 43
Induced human pluripotent stem cells: promises and open questions
Rolletschek, A., & Wobus, A. M. (2009) · Biological Chemistry · doi:10.1515/bc.2009.103 · Cited by 45
“However, other applications of iPS cells are also feasible, such as the use of iPS cells to study pathomechanisms of diseases, including Parkinson’s disease and Huntington’s disease, juvenile-onset type 1 diabetes mellitus or amyotrophic lateral sclerosis (Dimos et al., 2008; Park et al., 2008).”
BackgroundBasal Forebrain Cholinergic Neurons: Linking Down Syndrome and Alzheimer’s Disease
Martinez, J. L., Zammit, M. D., West, N. R., Christian, B. T., & Bhattacharyya, A. (2021) · Frontiers in Aging Neuroscience · doi:10.3389/fnagi.2021.703876 · Cited by 47
Chen, Y., Chen, J., Sun, X., Yu, J., Qian, Z. Y., Wu, L., Xu, X., Wan, X., Jiang, Y., Zhang, J., et al. (2020) · Aging Cell · doi:10.1111/acel.13185 · Cited by 52
“…(iPSCs) derived from autologous somatic cells transduced with Yamanaka factors can be subsequently differentiated into desired cell types in vitro and further applied to treat age-related diseases in a variety of types of tissues (Park et al., 2008; Si-Tayeb et al., 2010; Takebe et al., 2013).”
BackgroundKavitha, M. S., Kurita, T., Park, S.-Y., Chien, S., Bae, J., & Ahn, B. (2017) · PLoS ONE · doi:10.1371/journal.pone.0189974 · Cited by 60
Siller, R., Naumovska, E., Mathapati, S., Lycke, M., Greenhough, S., & Sullivan, G. (2016) · Scientific Reports · doi:10.1038/srep37178 · Cited by 43
Suzuki, S., Sargent, R. G., Illek, B., Fischer, H., Esmaeili-Shandiz, A., Yezzi, M. J., Lee, A., Yang, Y., Kim, S., Renz, P., et al. (2016) · Molecular Therapy: Nucleic Acids · doi:10.1038/mtna.2015.43 · Cited by 43
The evolving field of induced pluripotency: Recent progress and future challenges
Sommer, C., & Mostoslavsky, G. (2012) · Journal of Cellular Physiology · doi:10.1002/jcp.24155 · Cited by 46
Conforti, P., Camnasio, S., Mutti, C., Valenza, M., Thompson, M., Fossale, E., Zeitlin, S., MacDonald, M., Zuccato, C., & Cattaneo, E. (2013) · Neurobiology of Disease · doi:10.1016/j.nbd.2012.10.015 · Cited by 43
Chen, M., Zhang, H., Wu, J., Xu, L., Xu, D., Sun, J., He, Y., Zhou, X., Wang, Z., Wu, L., et al. (2012) · Biomaterials · doi:10.1016/j.biomaterials.2012.04.001 · Cited by 44
Mitochondrial dysfunction and Down's syndrome: Is there a role for coenzyme Q10?
Tiano, L., & Busciglio, J. A. (2011) · BioFactors · doi:10.1002/biof.184 · Cited by 45
Systems biology approaches to heart development and congenital heart disease
Sperling, S. (2011) · Cardiovascular Research · doi:10.1093/cvr/cvr126 · Cited by 48
“Besides the immediate use of patient-material, the generation of patient-specific iPSCs offers a new and very promising way to study human diseases.(67,68) Different groups have already shown the power of iPSCs for the analysis of congenital arrhythmia and malformation.”
BackgroundiPSCs-Based Neural 3D Systems: A Multidimensional Approach for Disease Modeling and Drug Discovery
Costamagna, G., Andreoli, L., Corti, S. P., & Faravelli, I. (2019) · Cells · doi:10.3390/cells8111438 · Cited by 40
“Moreover, iPSCs can be differentiated into cells able to recapitulate the hallmarks of pathological cells and tissues to develop disease models and test new potential therapies [13].”
BackgroundNeural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease
Vu, M., Li, R., Baskfield, A., Lu, B., Farkhondeh, A., Gorshkov, K., Motabar, O., Beers, J. K., Chen, G.-K., Zou, J., et al. (2018) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-018-0886-3 · Cited by 42
Sridhar, A., Ohlemacher, S. K., Langer, K. B., & Meyer, J. S. (2016) · Stem Cells Translational Medicine · doi:10.5966/sctm.2015-0093 · Cited by 42
Cardiac disease modeling using induced pluripotent stem cell-derived human cardiomyocytes
Dell’Era, P. (2015) · World Journal of Stem Cells · doi:10.4252/wjsc.v7.i2.329 · Cited by 45
Molecular beacon enabled purification of living cells by targeting cell-type specific mRNAs
Wile, B., Ban, K., Yoon, Y., & Bao, G. (2014) · Nature Protocols · doi:10.1038/nprot.2014.154 · Cited by 42
“There are five essential elements of the MB-based protocol for the isolation of a specific cell type from differentiating cells (Figure 2): (1) identification of specific mRNAs that are highly expressed only in the desired cell type (Figure 2A); (2) design and validation of MBs targeting specific mRNAs with high signa…”
MethodModeling Neurodevelopmental Disorders Using Human Pluripotent Stem Cells
Telias, M., & Ben‐Yosef, D. (2014) · Stem Cell Reviews and Reports · doi:10.1007/s12015-014-9507-2 · Cited by 43
“LNS hiPSCs were first established in 2008 [198], but no information on their neural potential was provided.”
JAK-STAT3 and somatic cell reprogramming
Tang, Y., & Tian, X. (2013) · JAK-STAT · doi:10.4161/jkst.24935 · Cited by 44
Lin, C.-Y., Peng, C.-Y., Huang, T.-T., Wu, M.-L., Lai, Y.-L., Peng, D., Chen, P.-F., Chen, H.-F., Yen, B., Wu, K. K., et al. (2012) · Stem Cells and Development · doi:10.1089/scd.2011.0304 · Cited by 42
“Several iPS cell lines with genetic mutations have been generated [42,43]; of these, Rett syndrome iPS cells-derived neurons were used to investigate the cellular and molecular mechanisms underlying the abnormalities of the disease [43].”
MethodTranslational Prospects for Human Induced Pluripotent Stem Cells
CSETE, M. E. (2010) · Regenerative Medicine · doi:10.2217/rme.10.39 · Cited by 43
Induced pluripotent stem cell therapies for retinal disease
Comyn, O., Lee, E. J., & MacLaren, R. (2010) · Current Opinion in Neurology · doi:10.1097/wco.0b013e3283352f96 · Cited by 42
Umbilical Cord Transplantation: Epilogue
Broxmeyer, H. (2010) · Seminars in hematology (Print) · doi:10.1053/j.seminhematol.2009.10.002 · Cited by 42
Hepatocyte-like cells derived from induced pluripotent stem cells
Roy‐Chowdhury, N., Wang, X., Guha, C., & Roy‐Chowdhury, J. (2016) · Hepatology International · doi:10.1007/s12072-016-9757-y · Cited by 45
Wada, H., Kojo, S., Kusama, C., Okamoto, N., Sato, Y., Ishizuka, B., & Seino, K. (2011) · International Immunology · doi:10.1093/intimm/dxq458 · Cited by 42
Early Actions of Neurotransmitters During Cortex Development and Maturation of Reprogrammed Neurons
Ojeda, J., & Ávila, A. (2019) · Frontiers in Synaptic Neuroscience · doi:10.3389/fnsyn.2019.00033 · Cited by 48
Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic
Miniarikova, J., Evers, M. M., & Konstantinova, P. S. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.02.002 · Cited by 43
G Protein‐Coupled Receptors and Adipogenesis: A Focus on Adenosine Receptors
Eisenstein, A. S., & Ravid, K. (2013) · Journal of Cellular Physiology · doi:10.1002/jcp.24473 · Cited by 44
“QRFP is a member of the RFamide family of peptides which all contain carboxy- JOURNAL OF CELLULAR PHYSIOLOGY terminal arginine (R) and amidated phenylalanine (F) residues (Price and Greenberg, 1977).”
BackgroundCell therapy in critical limb ischemia: current developments and future progress.
Mamidi, M., Pal, R., Dey, S., Abdullah, B. J. J. B., Zakaria, Z., Rao, M. S., & Das, A. (2012) · Cytotherapy · doi:10.3109/14653249.2012.693156 · Cited by 41
Modelling familial dysautonomia in human induced pluripotent stem cells
Lee, G., & Studer, L. (2011) · Philosophical Transactions of the Royal Society B Biological Sciences · doi:10.1098/rstb.2011.0026 · Cited by 38
Patient-Specific Pluripotent Stem Cells in Neurological Diseases
Durnaoglu, S., Genç, Ş., & Genç, K. (2011) · Stem Cells International · doi:10.4061/2011/212487 · Cited by 44
“The differentiation of human iPSCs including patient-specific iPSCs into neural ectoderm cells [82] has been demonstrated by positive staining for NES [83], increased expression of PAX6, and neural cell adhesion molecule mRNAs [31, 84].”
BackgroundInfluentialModeling Neurological Diseases Using Patient-Derived Induced Pluripotent Stem Cells
Juopperi, T. A., Song, H., & Ming, G. (2011) · Future Neurology · doi:10.2217/fnl.11.14 · Cited by 39
Wong, G. K. Y., & Chiu, A. T. (2010) · Biotechnology Advances · doi:10.1016/j.biotechadv.2010.07.005 · Cited by 37
Reprogramming of somatic cell identity.Preprint
Hanna, J. H., Carey, B. W., & Jaenisch, R. (2008) · Cold Spring Harbor Symposia on Quantitative Biology · doi:10.1101/sqb.2008.73.025 · Cited by 41
“Finally, pluripotent lines derived from human patients with specific diseases constitute invaluable tools and an unlimited source for biological material that can be used to study these complex diseases in the Petri dish (Dimos et al. 2008; Park et al. 2008a).”
BackgroundOvadia, E. M., Colby, D. W., & Kloxin, A. M. (2018) · Biomaterials Science · doi:10.1039/c8bm00099a · Cited by 40
Region Specific Brain Organoids to Study Neurodevelopmental Disorders
Susaimanickam, P. J., Kiral, F. R., & Park, I. (2022) · International Journal of Stem Cells · doi:10.15283/ijsc22006 · Cited by 47
“Disease specific iPSCs (6) have been used to obtain neurons, oligodendrocytes and micro-”
MethodSelf-citationOrtiz-Vitali, J. L., & Darabi, R. (2019) · Cells · doi:10.3390/cells8010020 · Cited by 51
“Initial reports in 2008 and later were mainly focused on the generation of iPSCs from MDs (such as DMD and LGMD) and proof of concept for their myogenic differentiation [28,29,30,31].”
BackgroundInduced Pluripotent Stem Cells for Duchenne Muscular Dystrophy Modeling and Therapy
Danišovič, L., Culenová, M., & Csobonyeiova, M. (2018) · Cells · doi:10.3390/cells7120253 · Cited by 40
“The same core set of factors has been used to prepare human iPSCs [18].”
BackgroundCRISPR/Cas9-mediated correction of human genetic disease
Men, K., Duan, X., He, Z., Yang, Y., Yao, S., & Wei, Y. (2017) · Science China Life Sciences · doi:10.1007/s11427-017-9032-4 · Cited by 44
Comprehensive Protocols for CRISPR/Cas9‐based Gene Editing in Human Pluripotent Stem Cells
Santos, D. P., Kiskinis, E., Eggan, K. C., & Merkle, F. T. (2016) · Current Protocols in Stem Cell Biology · doi:10.1002/cpsc.15 · Cited by 45
“, 1998) has been historically limited, but the advent of human induced pluripotent stem cells (hiPSC) (Nakagawa et al., 2008; Park et al., 2008; Takahashi et al., 2007; Yu et al., 2007) has enabled the wider research community to engage in hPSC research.”
BackgroundHuman dermal fibroblasts in psychiatry research
Kalman, J. L., Garbett, K. A., Janka, Z., & Mirnics, K. (2016) · Neuroscience · doi:10.1016/j.neuroscience.2016.01.067 · Cited by 45
“Disease- and patient-specific iPSC lines have been created for several disorders, including Parkinson’s disease (Park et al., 2008 and Soldner et al., 2011), amyotrophic lateral sclerosis (Dimos et al., 2008), spinal muscular atrophy (Ebert et al., 2009), Alzheimer’s disease (Israel et al., 2012),…”
BackgroundOstrovidov, S., Shi, X., Sadeghian, R. B., Salehi, S., Fujie, T., Bae, H., Ramalingam, M., & Khademhosseini, A. (2015) · Stem Cell Reviews and Reports · doi:10.1007/s12015-015-9618-4 · Cited by 44
“Moreover, it is possible to generate diseased-iPS cells for disease models and drug development or to make genetic corrections in these cells before myogenic differentiation and transplantation [59, 61, 167, 185].”
Fan, Y., Wu, J., Ashok, P., Hsiung, M., & Tzanakakis, E. (2014) · Stem Cell Reviews and Reports · doi:10.1007/s12015-014-9544-x · Cited by 40
“These cells also provide a means toward patient-specific therapies and disease model development [10, 11].”
Directed In Vitro Myogenesis of Human Embryonic Stem Cells and Their In Vivo Engraftment
Hwang, Y., Suk, S., Lin, S., Tierney, M. T., Du, B., Seo, T., Mitchell, A., Sacco, A., & Varghese, S. (2013) · PLoS ONE · doi:10.1371/journal.pone.0072023 · Cited by 40
“These qualities suggest widespread utility of hESCs in applications ranging from cell-based regenerative therapy to drug screening platforms [4,5,6].”
BackgroundCanfield, S. G., Šepac, A., Sedlić, F., Muravyeva, M., Bai, X., & Bosnjak, Z. J. (2012) · Anesthesiology · doi:10.1097/aln.0b013e3182655e96 · Cited by 39
Stem Cells asIn VitroModel of Parkinson's Disease
Martínez‐Morales, P., & Liste, I. (2012) · Stem Cells International · doi:10.1155/2012/980941 · Cited by 42
“The first reported lines of reprogrammed cells, termed induced pluripotent stem cells (iPSCs), were originally generated by introducing four transcription factors: Oct3/4, Sox2, Klf4, and c-myc (or Nanog, Lin28) into adult fibroblasts.”
BackgroundNeurogenic potential of progenitor cells isolated from postmortem human Parkinsonian brains
Wang, S., Okun, M. S., Suslov, O. N., Zheng, T., McFarland, N. R., Vedam‐Mai, V., Foote, K. D., Roper, S. N., Yachnis, A. T., Siebzehnrübl, F. A., et al. (2012) · Brain Research · doi:10.1016/j.brainres.2012.04.039 · Cited by 41
“Likewise, this cell could be compared with counterparts generated from normal, and PD induced pluripotent stem cells (iPSCs) and induced neurons/ dopamine neurons (Takahashi et al., 2007; Yu et al., 2007; Park et al., 2008; Nguyen et al., 2011; Pfisterer et al., 2011) in future studies attempting to categorize and sta…”
BackgroundTakayama, N., & Eto, K. (2012) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-012-0995-4 · Cited by 40
“Fourth, patient-specific iPSCs facilitate the study of disease mechanisms and drug screening [78].”
BackgroundShimada, H., Hashimoto, Y., Nakada, A., Shigeno, K., & Nakamura, T. (2012) · Biochemical and Biophysical Research Communications - BBRC · doi:10.1016/j.bbrc.2011.11.111 · Cited by 40
Efficient Allele-Specific Targeting of LRRK2 R1441 Mutations Mediated by RNAi
de Yñigo-Mojado, L., Martín‐Ruiz, I., & Sutherland, J. D. (2011) · PLoS ONE · doi:10.1371/journal.pone.0021352 · Cited by 40
“Thus, SM2-RG(11) is a good candidate to proceed into more physiological studies, perhaps in a recently described mouse model carrying the human R1441G LRRK2 allele [73], or in patient-derived cell lines made feasible by induced pluripotent stem cell technology [74].”
BackgroundThe Power of Mouse Genetics to Review Study Spermatogenesis
Yatsenko, A., Iwamori, N., Iwamori, T., & Matzuk, M. (2009) · Journal of Andrology · doi:10.2164/jandrol.109.008227 · Cited by 40
“conflicts about moral issue and immunotolerance ( Park, et al., 2008a ).”
Evolving toward a human-cell based and multiscale approach to drug discovery for CNS disorders
Schadt, E. E., Buchanan, S., Brennand, K. J., & Merchant, K. M. (2014) · Frontiers in Pharmacology · doi:10.3389/fphar.2014.00252 · Cited by 39
“Multimodal models can be used to identify disease signatures, by using the networks to organize the signatures according to the subnetworks (and the biological processes that they define), which are associated with disease (Schadt et al., 2005, 2008; Chen et al., 2008; Emilsson et al., 2008; Yang…”
MethodThe Use of Induced Pluripotent Stem Cells for the Study and Treatment of Liver Diseases
Hansel, M. C., Davila, J. C., Vosough, M., Gramignoli, R., Skvorak, K. J., Dorko, K., Marongiu, F., Blake, W. L., & Strom, S. C. (2016) · Current Protocols in Toxicology · doi:10.1002/0471140856.tx1413s67 · Cited by 36
Stanslowsky, N., Haase, A., Martin, U., Naujock, M., Leffler, A., Dengler, R., & Wegner, F. (2014) · Stem cell research & therapeutics · doi:10.1186/scrt423 · Cited by 39
Lau, T., Ho, L., & Wang, D.-A. (2013) · Biomaterials · doi:10.1016/j.biomaterials.2013.05.034 · Cited by 39
“The successful development of induced pluripotent stem cell (iPSC) technology [2,4,5] provides a potential solution to circumvent such limitations in the current cell-based therapies by generating patient-specific stem cells and associated progeny [3,6] for tissue replacement.”
BackgroundiPSCs and small molecules: a reciprocal effort towards better approaches for drug discovery
Zhang, R., Zhang, L., & Xie, X. (2013) · Acta Pharmacologica Sinica · doi:10.1038/aps.2013.21 · Cited by 42
Impact of new technologies for cellular screening along the drug value chain
Möller, C., & Slack, M. (2010) · Drug Discovery Today · doi:10.1016/j.drudis.2010.02.010 · Cited by 38
Wu, Z., Li, Y.-H., Huang, A. C. J., Li, M., Zhang, X., Wang, J.-G., Yang, M., & Chen, M. (2010) · Chinese Journal of Integrative Medicine · doi:10.1007/s11655-010-0041-z · Cited by 43
Induced Pluripotency of Mouse and Human Somatic CellsPreprint
Aherali, N. M., & Ochedlinger, K. H. (2008) · Cold Spring Harbor Symposia on Quantitative Biology · doi:10.1101/sqb.2008.73.017 · Cited by 31
“This, and the recent derivation of patient-specifc iPS cell lines from 11 different genetic disorders (Dimos et al. 2008; Park et al. 2008a), demonstrates the potential utility of reprogramming research in a therapeutic setting.”
BackgroundInfluentialSelf-citationLopes, C., Tang, Y., Anjo, S. I., Manadas, B. J., Onofre, I., de Almeida, L. P., Daley, G. Q., Schlaeger, T. M., & Rego, A. C. (2020) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2020.576592 · Cited by 40
Self-citationA Chemical Recipe for Generation of Clinical-Grade Striatal Neurons from hESCs
Wu, M.-H., Zhang, D., Bi, C.-Y., Mi, T.-W., Zhu, W., Xia, L., Teng, Z., Hu, B.-Y., & Wu, Y.-H. (2018) · Stem Cell Reports · doi:10.1016/j.stemcr.2018.08.005 · Cited by 38
“Currently many disease-specific hiPSCs suchasParkinson’sdisease,HD, andAlzheimer’s disease have been established (Park et al., 2008).”
BackgroundMaterials for Neural Differentiation, Trans‐Differentiation, and Modeling of Neurological Disease
Gong, L., Cao, L., Shen, Z., Shao, L., Gao, S., Zhang, C., Lü, J., & Li, W. (2018) · Advanced Materials · doi:10.1002/adma.201705684 · Cited by 40
Begum, A. N., Guoynes, C. D., Cho, J., Hao, J., Lutfy, K., & Hong, Y. (2015) · Stem Cell Research · doi:10.1016/j.scr.2015.10.014 · Cited by 43
“, 2009), as well as inherited and sporadic forms of various human neurodegenerative conditions, including motor neuron disease, Niemann-Pick disease (NPD), Huntington disease (HD), Parkinson's disease (PD) and Alzheimer's disease (AD) (Dimos et al., 2008; Park et al., 2008; Yagi et al., 2011; Shi et al., 2012a; Israel…”
BackgroundInfluentialCanals, I., Soriano, J., Orlandi, J. G., Torrent, R., Richaud-Patin, Y., Jiménez-Delgado, S., Merlin, S., Follenzi, A., Consiglio, A., Vilageliu, L., et al. (2015) · Stem Cell Reports · doi:10.1016/j.stemcr.2015.08.016 · Cited by 38
Johnson-Kerner, B., Ahmad, F. S., Diaz, A. G., Greene, J. P., Gray, S. J., Samulski, R. J., Chung, W. K., Coster, R. V., Maertens, P. M., Noggle, S., et al. (2014) · Human Molecular Genetics · doi:10.1093/hmg/ddu556 · Cited by 43
“Many studies using iPSC models from patients and controls have left open the question as to whether the observed differences are true disease-relevant phenotypes or rather reflect inter-individual genetic variability not directly related to disease (29).”
BackgroundLiu, Y., Xue, Y., Ridley, S., Zhang, D., Rezvani, K. S., Fu, X.-D., & Wang, H. (2014) · PLoS ONE · doi:10.1371/journal.pone.0109621 · Cited by 37
Recent technological updates and clinical applications of induced pluripotent stem cells
Diecke, S., Jung, S. M., Lee, J., & Ju, J. H. (2014) · The Korean Journal of Internal Medicine · doi:10.3904/kjim.2014.29.5.547 · Cited by 44
“Disease modeling with iPSCs recapitulates a pathologic condition in vitro by reprogramming a patient’s somatic cells into iPSCs, followed by redifferentiation of the patient-specific iPSCs into disease-specific cell types [52-60].”
MethodGupta, K., Chandran, S., & Hardingham, G. E. (2012) · British Journal of Clinical Pharmacology · doi:10.1111/bcp.12022 · Cited by 34
Pereira, M. C., Secco, M., Suzuki, D. E., Janjoppi, L., Rodini, C., Torres, L. B., Araujo, B., Cavalheiro, E., Zatz, M., & Okamoto, O. (2011) · Stem Cell Reviews and Reports · doi:10.1007/s12015-011-9256-4 · Cited by 38
“In addition to the potential development of teratomas after transplantation of pluripotent cells, in the case of genetically reprogrammed cells, it remains unclear whether iPS cells derived from PD patients would generate dysfunctional neurons due to inherited genetic alterations associated with the disease [17].”
BackgroundChallenges of using pluripotent stem cells for safety assessments of substances.
Vojnits, K., & Bremer, S. (2010) · Toxicology · doi:10.1016/j.tox.2009.12.003 · Cited by 38
Payne, N. L., Sylvain, A., O’Brien, C., Herszfeld, D., Sun, G., & Bernard, C. C. A. (2014) · New Biotechnology · doi:10.1016/j.nbt.2014.05.001 · Cited by 41
Ravi, I., Baunthiyal, M., & Saxena, J. (2014) · Springer India · doi:10.1007/978-81-322-1554-7 · Cited by 37
X Chromosome of Female Cells Shows Dynamic Changes in Status during Human Somatic Cell Reprogramming
Kim, K., Hysolli, E., Tanaka, Y., Wang, B., Jung, Y. W., Pan, X., Weissman, S. M., & Park, I. (2014) · Stem Cell Reports · doi:10.1016/j.stemcr.2014.04.003 · Cited by 40
“…and differentiation into cells of all three germ layers. iPSCs alsomaintain the genomic composition of parental somatic cells and thus are considered as autologous cellular resources that are critical for cell therapy and in vitro disease modeling (Park et al., 2008a;Wu andHochedlinger, 2011).”
BackgroundSelf-citationDing, D.-C., Lin, C.-H., Shyu, W., & Lin, S.-Z. (2013) · Cell Transplantation · doi:10.3727/096368912x655091 · Cited by 37
Alzheimer's Disease Modeling: Ups, Downs, and Perspectives for Human Induced Pluripotent Stem Cells
Wojda, U., & Kuźnicki, J. (2013) · Journal of Alzheimer s Disease · doi:10.3233/jad-121984 · Cited by 40
“Human somatic cells were then reprogrammed into human iPSCs (hiPSCs) using the same gene cocktail [213, 214].”
Human Pluripotent Stem Cells: Applications and Challenges in Neurological Diseases
Hibaoui, Y., & Féki, A. (2012) · Frontiers in Physiology · doi:10.3389/fphys.2012.00267 · Cited by 35
Lu, H.-E., Tsai, M., Yang, Y.-C., Yuan, C.-C., Wang, T., Lin, X., Tseng, C., & Hwang, S. (2011) · Experimental Cell Research · doi:10.1016/j.yexcr.2011.05.017 · Cited by 36
Induced Pluripotent Stem Cell Therapies for Geographic Atrophy of Age-Related Macular Degeneration
Du, H., Lim, S., Grob, S., & Zhang, K. (2011) · Seminars in Ophthalmology · doi:10.3109/08820538.2011.577498 · Cited by 38
Neurodegenerative disease-specific induced pluripotent stem cell research
Inoue, H. (2010) · Experimental Cell Research · doi:10.1016/j.yexcr.2010.04.022 · Cited by 29
Applications of 3D Bioprinting Technology in Induced Pluripotent Stem Cells-Based Tissue Engineering
Shukla, A., Gao, G., & Kim, B. (2022) · Micromachines · doi:10.3390/mi13020155 · Cited by 36
Human iPSCs and Genome Editing Technologies for Precision Cardiovascular Tissue Engineering
Gähwiler, E. K. N., Motta, S. E., Martin, M., Nugraha, B., Hoerstrup, S. P., & Emmert, M. Y. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2021.639699 · Cited by 42
“Among the existing SC therapies, hiPSCs emerge as a potential cell source for CVD modeling and treatment (Park and Yoon, 2018; Sadahiro, 2019; Parrotta et al., 2020).”
BackgroundDysfunction of iPSC-derived endothelial cells in human Hutchinson–Gilford progeria syndrome
Matrone, G., Thandavarayan, R., Walther, B. K., Meng, S., Mojiri, A., & Cooke, J. (2019) · Cell Cycle · doi:10.1080/15384101.2019.1651587 · Cited by 36
Okumura, T., Horie, Y., Lai, C., Lin, H.-T., Shoda, H., Natsumoto, B., Fujio, K., Eri, K., Okano, T., Ono, S., et al. (2019) · Stem Cell Research & Therapy · doi:10.1186/s13287-019-1273-2 · Cited by 40
Modeling Cancer with Pluripotent Stem Cells
Gingold, J. A., Zhou, R., Lemischka, I. R., & Lee, D. (2016) · Trends in cancer · doi:10.1016/j.trecan.2016.07.007 · Cited by 35
Cellular Therapies for Muscular Dystrophies: Frustrations and Clinical Successes
Negroni, E., Bigot, A., Butler-Browne, G., Trollet, C., & Mouly, V. (2016) · Human Gene Therapy · doi:10.1089/hum.2015.139 · Cited by 36
Kim, K. H., Liu, J., Galvin, R. J. S., Dage, J. L., Egeland, J. A., Smith, R. C., Merchant, K., & Paul, S. M. (2015) · PLoS ONE · doi:10.1371/journal.pone.0142693 · Cited by 39
“Immunocytological analysis of iPSCs and embryoid bodies was as described previously [30].”
BackgroundMethodiPSC-based drug screening for Huntington׳s disease
Zhang, N., Bailus, B. J., Ring, K. L., & Ellerby, L. (2015) · Brain Research · doi:10.1016/j.brainres.2015.09.020 · Cited by 37
“generated a group of human iPSC lines from patients with various diseases including HD (31).”
BackgroundInvited review: Stem cells and muscle diseases: advances in cell therapy strategies
Négroni, E., Gidaro, T., Bigot, A., Butler-Browne, G. S., Mouly, V., & Trollet, C. (2014) · Neuropathology and Applied Neurobiology · doi:10.1111/nan.12198 · Cited by 49
Induced Pluripotent Stem Cell Potential in Medicine, Specifically Focused on Reproductive Medicine
Botman, O., & Wyns, C. (2014) · Frontiers in Surgery · doi:10.3389/fsurg.2014.00005 · Cited by 41
The benefits and risks of stem cell technology
Leventhal, A., Chen, G.-B., Negro, A., & Boehm, M. (2011) · Oral Diseases · doi:10.1111/j.1601-0825.2011.01870.x · Cited by 36
“…lateral sclerosis), hematologic (including Fanconi's anemia and dyskeratosis congenital), and cardiac disorders (most notably the long QT syndrome) (Park et al, 2008). iPS cells from patients with the long QT syndrome are particularly interesting as they may provide an excellent platform for…”
BackgroundHuman neural stem cells: a model system for the study of Lesch-Nyhan disease neurological aspects.
Cristini, S., Navone, S., Canzi, L., Acerbi, F., Ciusani, E., Hladnik, U., de Gemmis, P., Alessandri, G., Colombo, A., Parati, E., et al. (2010) · Human Molecular Genetics · doi:10.1093/hmg/ddq072 · Cited by 36
“Park and colleagues (15) generated an iPS line from a female carrier of Lesch–Nyhan syndrome (LNSc-iPS2) that will be a valuable resource for studies of homologous recombination in iPS cells, and for analysis of X chromosome reactivation during reprogramming and random inactivation with differentiation.”
BackgroundCurrent progress and prospects of induced pluripotent stem cells
Chen, L., & Liu, L. (2009) · Science in China Series C Life Sciences · doi:10.1007/s11427-009-0092-6 · Cited by 33
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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