Creating Patient-Specific Neural Cells for the in Vitro Study of Brain Disorders

Stem Cell Reports

Volumn 5, Issue 6, 2015, Pages 933-945

  Brennand, Kristen J ^a   Marchetto, M Carol ^b   Benvenisty, Nissim ^c   Brüstle, Oliver ^d   Ebert, Allison ^e   Izpisua Belmonte, Juan Carlos ^b   Kaykas, Ajamete ^f   Lancaster, Madeline A ^g   Livesey, Frederick J ^h   McConnell, Michael J ⁱ   McKay, Ronald D ^j   Morrow, Eric M ^k   Muotri, Alysson R ^l   Panchision, David M ^m   Rubin, Lee L ⁿ   Sawa, Akira ^o   Soldner, Frank ^p   Song, Hongjun ^q   Studer, Lorenz ^r   Temple, Sally ^s   Vaccarino, Flora M ^t   Wu, Jun ^b   Vanderhaeghen, Pierre ^u   Gage, Fred H ^b   Jaenisch, Rudolf ^p   more..

^a MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^b SALK INSTITUTE FOR BIOLOGICAL STUDIES   (United States)

^c HEBREW UNIVERSITY OF JERUSALEM   (Israel)

^d UNIVERSITY OF BONN   (Germany)

^e MEDICAL COLLEGE OF WISCONSIN   (United States)

^f NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH   (United States)

^g MRC LABORATORY OF MOLECULAR BIOLOGY   (United Kingdom)

^h UNIVERSITY OF CAMBRIDGE   (United Kingdom)

ⁱ UNIVERSITY OF VIRGINIA   (United States)

^j LIEBER INSTITUTE FOR BRAIN DEVELOPMENT   (United States)

^k BROWN UNIVERSITY   (United States)

^l UNIVERSITY OF CALIFORNIA   (United States)

^m NATIONAL INSTITUTE OF MENTAL HEALTH   (United States)

ⁿ Harvard University   (United States)

^o JOHNS HOPKINS HOSPITAL   (United States)

^p WHITEHEAD INSTITUTE FOR BIOMEDICAL RESEARCH   (United States)

^q JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^r MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^s Neural Stem Cell Institute   (United States)

^t YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^u UNIVERSITÉ LIBRE DE BRUXELLES   (Belgium)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ASTROCYTE; BIPOLAR DISORDER; BLASTOCYST; BRAIN DISEASE; BRAIN TISSUE; CONFERENCE PAPER; ECTODERM; EMBRYONIC STEM CELL; GENE EXPRESSION; GENETIC POLYMORPHISM; GERM LAYER; HUMAN; IN VITRO STUDY; MITOCHONDRION; MOOD DISORDER; MOSAICISM; MOTONEURON; NERVE CELL; NERVOUS SYSTEM DEVELOPMENT; NEURAL STEM CELL; NONHUMAN; NUCLEAR REPROGRAMMING; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; SINGLE CELL ANALYSIS; SPINAL MUSCULAR ATROPHY; TRANSGENE; BRAIN; BRAIN DISEASES; CYTOLOGY; DRUG DEVELOPMENT; DRUG EFFECTS; GENETICS; INDUCED PLURIPOTENT STEM CELL; METABOLISM; PATHOLOGY; PATHOPHYSIOLOGY; PERSONALIZED MEDICINE; PROCEDURES;

BRAIN; BRAIN DISEASES; DRUG DISCOVERY; HUMANS; INDUCED PLURIPOTENT STEM CELLS; MOSAICISM; NEUROGENESIS; PRECISION MEDICINE;

EID: 84949526040     PISSN: 22136711     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.stemcr.2015.10.011     Document Type: Conference Paper

References (62)

1. A. Abyzov, J. Mariani, D. Palejev, Y. Zhang, M.S. Haney, L. Tomasini, A.F. Ferrandino, L.A. Rosenberg Belmaker, A. Szekely, M. Wilson, and et al. Somatic copy number mosaicism in human skin revealed by induced pluripotent stem cells Nature 492 2012 438 442
2. A. Adamo, S. Atashpaz, P.L. Germain, M. Zanella, G. D'Agostino, V. Albertin, J. Chenoweth, L. Micale, C. Fusco, C. Unger, and et al. 7q11.23 dosage-dependent dysregulation in human pluripotent stem cells affects transcriptional programs in disease-relevant lineages Nat. Genet. 47 2015 132 141
3. American Psychiatric Association. Diagnostic and Statistical Manual of Mental Disorders: DSM-IV Fourth Edition 1994 American Psychiatric Press
4. M. Benevento, P.D. Tonge, M.C. Puri, S.M. Hussein, N. Cloonan, D.L. Wood, S.M. Grimmond, A. Nagy, J. Munoz, and A.J. Heck Proteome adaptation in cell reprogramming proceeds via distinct transcriptional networks Nat. Commun. 5 2014 5613
5. K.J. Brennand, A. Simone, J. Jou, C. Gelboin-Burkhart, N. Tran, S. Sangar, Y. Li, Y. Mu, G. Chen, D. Yu, and et al. Modelling schizophrenia using human induced pluripotent stem cells Nature 473 2011 221 225
6. K. Brennand, J.N. Savas, Y. Kim, N. Tran, A. Simone, K. Hashimoto-Torii, K.G. Beaumont, H.J. Kim, A. Topol, I. Ladran, and et al. Phenotypic differences in hiPSC NPCs derived from patients with schizophrenia Mol. Psychiatry 20 2015 361 368
7. I.G. Brons, L.E. Smithers, M.W. Trotter, P. Rugg-Gunn, B. Sun, S.M. Chuva de Sousa Lopes, S.K. Howlett, A. Clarkson, L. Ahrlund-Richter, R.A. Pedersen, and L. Vallier Derivation of pluripotent epiblast stem cells from mammalian embryos Nature 448 2007 191 195
8. S.M. Chambers, Y. Mica, G. Lee, L. Studer, and M.J. Tomishima Dual-SMAD inhibition/WNT activation-based methods to induce neural crest and derivatives from human pluripotent stem cells Methods Mol. Biol. 1307 2013 329 343
9. Y.S. Chan, J. Göke, J.H. Ng, X. Lu, K.A. Gonzales, C.P. Tan, W.Q. Tng, Z.Z. Hong, Y.S. Lim, and H.H. Ng Induction of a human pluripotent state with distinct regulatory circuitry that resembles preimplantation epiblast Cell Stem Cell 13 2013 663 675
10. K.G. Chen, B.S. Mallon, K.R. Johnson, R.S. Hamilton, R.D. McKay, and P.G. Robey Developmental insights from early mammalian embryos and core signaling pathways that influence human pluripotent cell growth and differentiation Stem Cell Res. (Amst.) 12 2014 610 621
11. C.H. Chiang, Y. Su, Z. Wen, N. Yoritomo, C.A. Ross, R.L. Margolis, H. Song, and G.L. Ming Integration-free induced pluripotent stem cells derived from schizophrenia patients with a DISC1 mutation Mol. Psychiatry 16 2011 358 360
12. J.L. Clancy, H.R. Patel, S.M. Hussein, P.D. Tonge, N. Cloonan, A.J. Corso, M. Li, D.S. Lee, J.Y. Shin, J.J. Wong, and et al. Small RNA changes en route to distinct cellular states of induced pluripotency Nat. Commun. 5 2014 5522
13. A.D. Ebert, J. Yu, F.F. Rose Jr.; V.B. Mattis, C.L. Lorson, J.A. Thomson, and C.N. Svendsen Induced pluripotent stem cells from a spinal muscular atrophy patient Nature 457 2009 277 280
14. R. Eglen, and T. Reisine Primary cells and stem cells in drug discovery: emerging tools for high-throughput screening Assay Drug Dev. Technol. 9 2011 108 124
15. M. Eiraku, K. Watanabe, M. Matsuo-Takasaki, M. Kawada, S. Yonemura, M. Matsumura, T. Wataya, A. Nishiyama, K. Muguruma, and Y. Sasai Self-organized formation of polarized cortical tissues from ESCs and its active manipulation by extrinsic signals Cell Stem Cell 3 2008 519 532
16. L. Emdad, S.L. D'Souza, H.P. Kothari, Z.A. Qadeer, and I.M. Germano Efficient differentiation of human embryonic and induced pluripotent stem cells into functional astrocytes Stem Cells Dev. 21 2012 404 410
17. I. Espuny-Camacho, K.A. Michelsen, D. Gall, D. Linaro, A. Hasche, J. Bonnefont, C. Bali, D. Orduz, A. Bilheu, A. Herpoel, and et al. Pyramidal neurons derived from human pluripotent stem cells integrate efficiently into mouse brain circuits in vivo Neuron 77 2013 440 456
18. O. Gafni, L. Weinberger, A.A. Mansour, Y.S. Manor, E. Chomsky, D. Ben-Yosef, Y. Kalma, S. Viukov, I. Maza, A. Zviran, and et al. Derivation of novel human ground state naive pluripotent stem cells Nature 504 2013 282 286
19. R. Gorris, J. Fischer, K.L. Erwes, J. Kesavan, D.A. Peterson, M. Alexander, M.M. Nöthen, M. Peitz, T. Quandel, M. Karus, and O. Brüstle Pluripotent stem cell-derived radial glia-like cells as stable intermediate for efficient generation of human oligodendrocytes Glia 63 2015 2152 2167
20. T. Kadoshima, H. Sakaguchi, T. Nakano, M. Soen, S. Ando, M. Eiraku, and Y. Sasai Self-organization of axial polarity, inside-out layer pattern, and species-specific progenitor dynamics in human ES cell-derived neocortex Proc. Natl. Acad. Sci. USA 110 2013 20284 20289
21. M. Karus, S. Blaess, and O. Brüstle Self-organization of neural tissue architectures from pluripotent stem cells J. Comp. Neurol. 522 2014 2831 2844
22. P. Koch, P. Breuer, M. Peitz, J. Jungverdorben, J. Kesavan, D. Poppe, J. Doerr, J. Ladewig, J. Mertens, T. Tüting, and et al. Excitation-induced ataxin-3 aggregation in neurons from patients with Machado-Joseph disease Nature 480 2011 543 546
23. R. Krencik, J.P. Weick, Y. Liu, Z.J. Zhang, and S.C. Zhang Specification of transplantable astroglial subtypes from human pluripotent stem cells Nat. Biotechnol. 29 2011 528 534
24. M.A. Lancaster, M. Renner, C.A. Martin, D. Wenzel, L.S. Bicknell, M.E. Hurles, T. Homfray, J.M. Penninger, A.P. Jackson, and J.A. Knoblich Cerebral organoids model human brain development and microcephaly Nature 501 2013 373 379
25. D.S. Lee, J.Y. Shin, P.D. Tonge, M.C. Puri, S. Lee, H. Park, W.C. Lee, S.M. Hussein, T. Bleazard, J.Y. Yun, and et al. An epigenomic roadmap to induced pluripotency reveals DNA methylation as a reprogramming modulator Nat. Commun. 5 2014 5619
26. M.C. Marchetto, C. Carromeu, A. Acab, D. Yu, G.W. Yeo, Y. Mu, G. Chen, F.H. Gage, and A.R. Muotri A model for neural development and treatment of Rett syndrome using human induced pluripotent stem cells Cell 143 2010 527 539
27. J. Mariani, M.V. Simonini, D. Palejev, L. Tomasini, G. Coppola, A.M. Szekely, T.L. Horvath, and F.M. Vaccarino Modeling human cortical development in vitro using induced pluripotent stem cells Proc. Natl. Acad. Sci. USA 109 2012 12770 12775
28. J. Mariani, G. Coppola, P. Zhang, A. Abyzov, L. Provini, L. Tomasini, M. Amenduni, A. Szekely, D. Palejev, M. Wilson, and et al. FOXG1-Dependent Dysregulation of GABA/Glutamate Neuron Differentiation in Autism Spectrum Disorders Cell 162 2015 375 390
29. P.A. Marinho, T. Chailangkarn, and A.R. Muotri Systematic optimization of human pluripotent stem cells media using Design of Experiments Sci. Rep. 5 2015 9834
30. H. Marks, T. Kalkan, R. Menafra, S. Denissov, K. Jones, H. Hofemeister, J. Nichols, A. Kranz, A.F. Stewart, A. Smith, and H.G. Stunnenberg The transcriptional and epigenomic foundations of ground state pluripotency Cell 149 2012 590 604
31. M.J. McConnell, M.R. Lindberg, K.J. Brennand, J.C. Piper, T. Voet, C. Cowing-Zitron, S. Shumilina, R.S. Lasken, J.R. Vermeesch, I.M. Hall, and F.H. Gage Mosaic copy number variation in human neurons Science 342 2013 632 637
32. J. Mertens, K. Stüber, P. Wunderlich, J. Ladewig, J.C. Kesavan, R. Vandenberghe, M. Vandenbulcke, P. van Damme, J. Walter, O. Brüstle, and P. Koch APP processing in human pluripotent stem cell-derived neurons is resistant to NSAID-based γ-secretase modulation Stem Cell Reports 1 2013 491 498
33. J.D. Miller, Y.M. Ganat, S. Kishinevsky, R.L. Bowman, B. Liu, E.Y. Tu, P.K. Mandal, E. Vera, J.W. Shim, S. Kriks, and et al. Human iPSC-based modeling of late-onset disease via progerin-induced aging Cell Stem Cell 13 2013 691 705
34. S. Moore, L.D. Evans, T. Andersson, E. Portelius, J. Smith, T.B. Dias, N. Saurat, A. McGlade, P. Kirwan, K. Blennow, and et al. APP metabolism regulates tau proteostasis in human cerebral cortex neurons Cell Rep. 11 2015 689 696
35. A. Mullard 2014 FDA drug approvals Nat. Rev. Drug Discov. 14 2015 77 81
36. A.R. Muotri, K. Nakashima, N. Toni, V.M. Sandler, and F.H. Gage Development of functional human embryonic stem cell-derived neurons in mouse brain Proc. Natl. Acad. Sci. USA 102 2005 18644 18648
37. F. Nagashima, I.K. Suzuki, A. Shitamukai, H. Sakaguchi, M. Iwashita, T. Kobayashi, S. Tone, K. Toida, P. Vanderhaeghen, and Y. Kosodo Novel and robust transplantation reveals the acquisition of polarized processes by cortical cells derived from mouse and human pluripotent stem cells Stem Cells Dev. 23 2014 2129 2142
38. D.M. Panchision Meeting report: using stem cells for biological and therapeutics discovery in mental illness, April 2012 Stem Cells Transl. Med. 2 2013 217 222
39. M.F. Pescosolido, D.M. Stein, M. Schmidt, C.M. El Achkar, M. Sabbagh, J.M. Rogg, U. Tantravahi, R.L. McLean, J.S. Liu, A. Poduri, and E.M. Morrow Genetic and phenotypic diversity of NHE6 mutations in Christianson syndrome Ann. Neurol. 76 2014 581 593
40. C.W. Pouton, and J.M. Haynes Pharmaceutical applications of embryonic stem cells Adv. Drug Deliv. Rev. 57 2005 1918 1934
41. N. Renier, Z. Wu, D.J. Simon, J. Yang, P. Ariel, and M. Tessier-Lavigne iDISCO: a simple, rapid method to immunolabel large tissue samples for volume imaging Cell 159 2014 896 910
42. D. Sareen, A.D. Ebert, B.M. Heins, J.V. McGivern, L. Ornelas, and C.N. Svendsen Inhibition of apoptosis blocks human motor neuron cell death in a stem cell model of spinal muscular atrophy PLoS ONE 7 2012 e39113
43. J.W. Scannell, A. Blanckley, H. Boldon, and B. Warrington Diagnosing the decline in pharmaceutical R&D efficiency Nat. Rev. Drug Discov. 11 2012 191 200
44. A. Serio, B. Bilican, S.J. Barmada, D.M. Ando, C. Zhao, R. Siller, K. Burr, G. Haghi, D. Story, A.L. Nishimura, and et al. Astrocyte pathology and the absence of non-cell autonomy in an induced pluripotent stem cell model of TDP-43 proteinopathy Proc. Natl. Acad. Sci. USA 110 2013 4697 4702
45. Y. Shi, P. Kirwan, J. Smith, G. MacLean, S.H. Orkin, and F.J. Livesey A human stem cell model of early Alzheimer's disease pathology in Down syndrome Sci. Transl. Med. 4 2012 124ra29
46. F. Soldner, J. Laganière, A.W. Cheng, D. Hockemeyer, Q. Gao, R. Alagappan, V. Khurana, L.I. Golbe, R.H. Myers, S. Lindquist, and et al. Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations Cell 146 2011 318 331
47. M. Stadtfeld, and K. Hochedlinger Induced pluripotency: history, mechanisms, and applications Genes Dev. 24 2010 2239 2263
48. B.V. Stanzel, Z. Liu, S. Somboonthanakij, W. Wongsawad, R. Brinken, N. Eter, B. Corneo, F.G. Holz, S. Temple, J.H. Stern, and T.A. Blenkinsop Human RPE stem cells grown into polarized RPE monolayers on a polyester matrix are maintained after grafting into rabbit subretinal space Stem Cell Reports 2 2014 64 77
49. Y. Stelzer, O. Yanuka, and N. Benvenisty Global analysis of parental imprinting in human parthenogenetic induced pluripotent stem cells Nat. Struct. Mol. Biol. 18 2011 735 741
50. Y. Stelzer, I. Sagi, O. Yanuka, R. Eiges, and N. Benvenisty The noncoding RNA IPW regulates the imprinted DLK1-DIO3 locus in an induced pluripotent stem cell model of Prader-Willi syndrome Nat. Genet. 46 2014 551 557
51. Y. Stelzer, S. Bar, O. Bartok, S. Afik, D. Ronen, S. Kadener, and N. Benvenisty Differentiation of human parthenogenetic pluripotent stem cells reveals multiple tissue- and isoform-specific imprinted transcripts Cell Rep. 11 2015 308 320
52. K. Takahashi, K. Tanabe, M. Ohnuki, M. Narita, T. Ichisaka, K. Tomoda, and S. Yamanaka Induction of pluripotent stem cells from adult human fibroblasts by defined factors Cell 131 2007 861 872
53. Y. Takashima, G. Guo, R. Loos, J. Nichols, G. Ficz, F. Krueger, D. Oxley, F. Santos, J. Clarke, W. Mansfield, and et al. Resetting transcription factor control circuitry toward ground-state pluripotency in human Cell 158 2014 1254 1269
54. T.W. Theunissen, B.E. Powell, H. Wang, M. Mitalipova, D.A. Faddah, J. Reddy, Z.P. Fan, D. Maetzel, K. Ganz, L. Shi, and et al. Systematic identification of culture conditions for induction and maintenance of naive human pluripotency Cell Stem Cell 15 2014 471 487
55. P.D. Tonge, A.J. Corso, C. Monetti, S.M. Hussein, M.C. Puri, I.P. Michael, M. Li, D.S. Lee, J.C. Mar, N. Cloonan, and et al. Divergent reprogramming routes lead to alternative stem-cell states Nature 516 2014 192 197
56. A. Topol, S. Zhu, N. Tran, A. Simone, G. Fang, and K. Brennand Altered WNT signaling human induced pluripotent stem cell neural progenitor cells derived from four schizophrenia patients Biol. Psychiatry 78 2015 e29 e34
57. B. Valamehr, M. Robinson, R. Abujarour, B. Rezner, F. Vranceanu, T. Le, A. Medcalf, T.T. Lee, M. Fitch, D. Robbins, and P. Flynn Platform for induction and maintenance of transgene-free hiPSCs resembling ground state pluripotent stem cells Stem Cell Reports 2 2014 366 381
58. C.B. Ware, A.M. Nelson, B. Mecham, J. Hesson, W. Zhou, E.C. Jonlin, A.J. Jimenez-Caliani, X. Deng, C. Cavanaugh, S. Cook, and et al. Derivation of naive human embryonic stem cells Proc. Natl. Acad. Sci. USA 111 2014 4484 4489
59. Z. Wen, H.N. Nguyen, Z. Guo, M.A. Lalli, X. Wang, Y. Su, N.S. Kim, K.J. Yoon, J. Shin, C. Zhang, and et al. Synaptic dysregulation in a human iPS cell model of mental disorders Nature 515 2014 414 418
60. J. Wu, D. Okamura, M. Li, K. Suzuki, C. Luo, L. Ma, Y. He, Z. Li, C. Benner, I. Tamura, and et al. An alternative pluripotent state confers interspecies chimaeric competency Nature 521 2015 316 321
61. Q.L. Ying, J. Wray, J. Nichols, L. Batlle-Morera, B. Doble, J. Woodgett, P. Cohen, and A. Smith The ground state of embryonic stem cell self-renewal Nature 453 2008 519 523
62. D.X. Yu, F.P. Di Giorgio, J. Yao, M.C. Marchetto, K. Brennand, R. Wright, A. Mei, L. McHenry, D. Lisuk, J.M. Grasmick, and et al. Modeling hippocampal neurogenesis using human pluripotent stem cells Stem Cell Reports 2 2014 295 310