IPS cell derived neuronal cells for drug discovery

Trends in Pharmacological Sciences

Volumn 35, Issue 10, 2014, Pages 510-519

  Heilker, Ralf ^a   Traub, Stefanie ^a   Reinhardt, Peter ^b   Schöler, Hans R ^b,c   Sterneckert, Jared ^b  

^a BOEHRINGER INGELHEIM PHARMA GMBH AND CO KG   (Germany)

^b MAX PLANCK INSTITUTE FOR MOLECULAR BIOMEDICINE   (Germany)

^c UNIVERSITY OF MÜNSTER   (Germany)

Author keywords

disease modeling drug discovery; expandable neural progenitors; high content screening high throughput RT PCR; induced pluripotent stem (iPS) cells; phenotypic screening

Indexed keywords

AMPLIFIED LUMINESCENT PROXIMITY HOMOGENEOUS ASSAY; BIOASSAY; CELL COUNT; CELL EXPANSION; DRUG RESEARCH; DRUG SCREENING; DRUG TARGETING; HIGH THROUGHPUT SCREENING; HUMAN; INTRACELLULAR SIGNALING; NERVE CELL; NERVE CELL CULTURE; NERVE CELL DIFFERENTIATION; PLURIPOTENT STEM CELL; PROTEIN EXPRESSION; PROTEIN TARGETING; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; REVIEW; SENSITIVITY ANALYSIS; CYTOLOGY; DRUG DEVELOPMENT; DRUG EFFECTS; NEURAL STEM CELL; PROCEDURES;

DRUG DISCOVERY; DRUG EVALUATION, PRECLINICAL; HUMANS; INDUCED PLURIPOTENT STEM CELLS; NEURAL STEM CELLS; NEURONS;

EID: 84907894004     PISSN: 01656147     EISSN: 18733735     Source Type: Journal    
DOI: 10.1016/j.tips.2014.07.003     Document Type: Review

References (66)

1. E.H. Moors Towards a sustainable system of drug development Drug Discov. Today 2014 10.1016/j.drudis.2014.03.004
2. G.A. Fitzgerald Opinion: anticipating change in drug development: the emerging era of translational medicine and therapeutics Nat. Rev. Drug Discov. 4 2005 815 818
3. K. Takahashi, and S. Yamanaka Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors Cell 126 2006 663 676
4. H. Inoue iPS cells: a game changer for future medicine EMBO J. 33 2014 409 417
5. X. Xu Prevention of β-amyloid induced toxicity in human iPS cell-derived neurons by inhibition of Cyclin-dependent kinases and associated cell cycle events Stem Cell Res. 10 2013 213 227
6. D. McLaren Automated large-scale culture and medium-throughput chemical screen for modulators of proliferation and viability of human induced pluripotent stem cell-derived neuroepithelial-like stem cells J. Biomol. Screen. 18 2013 258 268
7. W. Sneader The discovery of aspirin: a reappraisal BMJ 321 2000 1591 1594
8. J.C. Dawson, and N.O. Carragher Quantitative phenotypic and pathway profiling guides rational drug combination strategies Front. Pharmacol. 5 2014 118
9. T.P. Kenakin, and D.A. Cook Blockade of histamine-induced contractions of guinea pig ileum by β-haloalkylamines Can. J. Physiol. Pharmacol. 54 1976 386 392
10. T.P. Kenakin A Pharmacology Primer 2009 Academic Press Elsevier
11. C. Ribas The G protein-coupled receptor kinase (GRK) interactome: role of GRKs in GPCR regulation and signaling Biochim. Biophys. Acta 1768 2007 913 922
12. J. Mertens APP processing in human pluripotent stem cell-derived neurons is resistant to NSAID-based γ-secretase modulation Stem Cell Rep. 1 2013 491 498
13. E.F. Ullman Luminescent oxygen channeling immunoassay: measurement of particle binding kinetics by chemiluminescence Proc. Natl. Acad. Sci. U.S.A. 91 1994 5426 5430
14. D.W. Young Integrating high-content screening and ligand-target prediction to identify mechanism of action Nat. Chem. Biol. 4 2008 59 68
15. F. Zanella High content screening: seeing is believing Trends Biotechnol. 28 2010 237 245
16. D.G. Healy Phenotype, genotype, and worldwide genetic penetrance of LRRK2-associated Parkinson's disease: a case-control study Lancet Neurol. 7 2008 583 590
17. T.B. Sherer Mechanism of toxicity in rotenone models of Parkinson's disease J. Neurosci. 23 2003 10756 10764
18. N. Ramsden Chemoproteomics-based design of potent LRRK2-selective lead compounds that attenuate Parkinson's disease-related toxicity in human neurons ACS Chem. Biol. 6 2011 1021 1028
19. X. Deng Leucine-rich repeat kinase 2 inhibitors: a patent review (2006-2011) Expert Opin. Ther. Pat. 22 2012 1415 1426
20. P. Reinhardt Genetic correction of a LRRK2 mutation in human iPSCs links parkinsonian neurodegeneration to ERK-dependent changes in gene expression Cell Stem Cell 12 2013 354 367
21. R.K. Saiki Enzymatic amplification of β-globin genomic sequences and restriction site analysis for diagnosis of sickle cell anemia Science 230 1985 1350 1354
22. J. Schlesinger Evaluation of the LightCycler 1536 instrument for high-throughput quantitative real-time PCR Methods 50 2010 S19 S22
23. L. Ghislain Sub-microliter Quantitative Real-time PCR from Whole Blood Using Acoustic Transfer With the Echo Liquid Handler 2013 Labcyte Ltd http://www.eventscribe.com/2012/posters/slas/ListView.asp
24. M.L. Metzker Sequencing technologies - the next generation Nat. Rev. Genet. 11 2010 31 46
25. K.J. Livak Oligonucleotides with fluorescent dyes at opposite ends provide a quenched probe system useful for detecting PCR product and nucleic acid hybridization PCR Methods Appl. 4 1995 357 362
26. P.M. Holland Detection of specific polymerase chain reaction product by utilizing the 5′-3′ exonuclease activity of Thermus aquaticus DNA polymerase Proc. Natl. Acad. Sci. U.S.A. 88 1991 7276 7280
27. G.L. Boulting A functionally characterized test set of human induced pluripotent stem cells Nat. Biotechnol. 29 2011 279 286
28. H.N. Nguyen LRRK2 mutant iPSC-derived DA neurons demonstrate increased susceptibility to oxidative stress Cell Stem Cell 8 2011 267 280
29. F.P. Di Giorgio Human embryonic stem cell-derived motor neurons are sensitive to the toxic effect of glial cells carrying an ALS-causing mutation Cell Stem Cell 3 2008 637 648
30. J. Altman, and G.D. Das Postnatal neurogenesis in the guinea-pig Nature 214 1967 1098 1101
31. L. Conti Niche-independent symmetrical self-renewal of a mammalian tissue stem cell PLoS Biol. 3 2005 e283
32. B.A. Reynolds, and S. Weiss Generation of neurons and astrocytes from isolated cells of the adult mammalian central nervous system Science 255 1992 1707 1710
33. M.S. Kaplan, and D.H. Bell Mitotic neuroblasts in the 9-day-old and 11-month-old rodent hippocampus J. Neurosci. 4 1984 1429 1441
34. 3H-thymidine-radiographic studies of neurogenesis in the rat olfactory bulb Exp. Brain Res. 50 1983 329 340
35. M.S. Kaplan, and J.W. Hinds Neurogenesis in the adult rat: electron microscopic analysis of light radioautographs Science 197 1977 1092 1094
36. C.B. Johansson Neural stem cells in the adult human brain Exp. Cell Res. 253 1999 733 736
37. P.S. Eriksson Neurogenesis in the adult human hippocampus Nat. Med. 4 1998 1313 1317
38. P. Koch A rosette-type, self-renewing human ES cell-derived neural stem cell with potential for in vitro instruction and synaptic integration Proc. Natl. Acad. Sci. U.S.A. 106 2009 3225 3230
39. S.C. Zhang In vitro differentiation of transplantable neural precursors from human embryonic stem cells Nat. Biotechnol. 19 2001 1129 1133
40. C.A. Fasano Efficient derivation of functional floor plate tissue from human embryonic stem cells Cell Stem Cell 6 2010 336 347
41. S.M. Chambers Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling Nat. Biotechnol. 27 2009 275 280
42. A.L. Perrier Derivation of midbrain dopamine neurons from human embryonic stem cells Proc. Natl. Acad. Sci. U.S.A. 101 2004 12543 12548
43. Y. Elkabetz Human ES cell-derived neural rosettes reveal a functionally distinct early neural stem cell stage Genes Dev. 22 2008 152 165
44. B.Y. Hu, and S.C. Zhang Differentiation of spinal motor neurons from pluripotent human stem cells Nat. Protoc. 4 2009 1295 1304
45. C.R. Nicholas Functional maturation of hPSC-derived forebrain interneurons requires an extended timeline and mimics human neural development Cell Stem Cell 12 2013 573 586
46. A.M. Maroof Directed differentiation and functional maturation of cortical interneurons from human embryonic stem cells Cell Stem Cell 12 2013 559 572
47. + medium-sized spiny neurons Development 140 2013 301 312
48. S. Kriks Dopamine neurons derived from human ES cells efficiently engraft in animal models of Parkinson's disease Nature 480 2011 547 551
49. A. Falk Capture of neuroepithelial-like stem cells from pluripotent stem cells provides a versatile system for in vitro production of human neurons PLoS ONE 7 2012 e29597
50. P. Koch Excitation-induced ataxin-3 aggregation in neurons from patients with Machado-Joseph disease Nature 480 2011 543 546
51. P. Koch Presenilin-1 L166P mutant human pluripotent stem cell-derived neurons exhibit partial loss of γ-secretase activity in endogenous amyloid-β generation Am. J. Pathol. 180 2012 2404 2416
52. P. Reinhardt Derivation and expansion using only small molecules of human neural progenitors for neurodegenerative disease modeling PLoS ONE 8 2013 e59252
53. W. Li Rapid induction and long-term self-renewal of primitive neural precursors from human embryonic stem cells by small molecule inhibitors Proc. Natl. Acad. Sci. U.S.A. 108 2011 8299 8304
54. G.H. Liu Progressive degeneration of human neural stem cells caused by pathogenic LRRK2 Nature 491 2012 603 607
55. C. Owen Chemokine receptors in airway disease: which receptors to target? Pulm. Pharmacol. Ther. 14 2001 193 292
56. J.A. Thomson Embryonic stem cell lines derived from human blastocysts Science 282 1998 1145 1147
57. B. Feng Reprogramming of fibroblasts into induced pluripotent stem cells with orphan nuclear receptor Esrrb Nat. Cell Biol. 11 2009 197 203
58. M. Nakagawa Generation of induced pluripotent stem cells without Myc from mouse and human fibroblasts Nat. Biotechnol. 26 2008 101 106
59. Y. Shi Induction of pluripotent stem cells from mouse embryonic fibroblasts by Oct4 and Klf4 with small-molecule compounds Cell Stem Cell 3 2008 568 574
60. J.B. Kim Pluripotent stem cells induced from adult neural stem cells by reprogramming with two factors Nature 454 2008 646 650
61. J.B. Kim Oct4-induced pluripotency in adult neural stem cells Cell 136 2009 411 419
62. J.B. Kim Direct reprogramming of human neural stem cells by OCT4 Nature 461 2009 649 653
63. S.D. Schwartz Embryonic stem cell trials for macular degeneration: a preliminary report Lancet 379 2012 713 720
64. Y. Shi A human stem cell model of early Alzheimer's disease pathology in Down syndrome Sci. Transl. Med. 4 2012 124ra129
65. M.A. Israel Probing sporadic and familial Alzheimer's disease using induced pluripotent stem cells Nature 482 2012 216 222
66. V. Luu-The Improved real-time RT-PCR method for high-throughput measurements using second derivative calculation and double correction Biotechniques 38 2005 287 293