Restoration of progranulin expression rescues cortical neuron generation in an induced pluripotent stem cell model of frontotemporal dementia

Stem Cell Reports

Volumn 4, Issue 1, 2015, Pages 16-24

  Raitano, Susanna ^a   Ordovàs, Laura ^a   De Muynck, Louis ^a,b   Guo, Wenting ^a,b   Espuny Camacho, Ira ^a,b,c   Geraerts, Martine ^a   Khurana, Satish ^a   Vanuytsel, Kim ^a   Tóth, Balazs I ^d   Voets, Thomas ^d   Vandenberghe, Rik ^a,g   Cathomen, Toni ^e   Van Den Bosch, Ludo ^a,b   Vanderhaeghen, Pierre ^a,b,c,f   Van Damme, Philip ^a,b,g   Verfaillie, Catherine M ^a  

^a UNIVERSITY OF LEUVEN   (Belgium)

^b DEPARTMENT OF PLANT SYSTEMS BIOLOGY   (Belgium)

^c UNIVERSITÉ LIBRE DE BRUXELLES   (Belgium)

^d LABORATORY OF MOLECULAR AND CELLULAR SIGNALING   (Belgium)

^e UNIVERSITY OF FREIBURG   (Germany)

^f Institute for Interdisciplinary Research ^*  (Belgium)

^g UNIVERSITY HOSPITALS LEUVEN   (Belgium)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; PROGRANULIN; TRANSCRIPTION FACTOR FOXP2; TRANSCRIPTOME; ZINC FINGER NUCLEASE; BIOLOGICAL MARKER; GRN PROTEIN, HUMAN; SIGNAL PEPTIDE;

ANIMAL CELL; ANIMAL TISSUE; ARTICLE; BRAIN CELL; BRAIN CORTEX; CELL DIFFERENTIATION; FRONTOTEMPORAL DEMENTIA; GENE EXPRESSION PROFILING; GENE LOCUS; GENE MUTATION; GENE TARGETING; HAPLOINSUFFICIENCY; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VITRO STUDY; MOTONEURON; MOUSE; NERVOUS SYSTEM DEVELOPMENT; NONHUMAN; PLURIPOTENT STEM CELL; PRIORITY JOURNAL; PROGENY; PROTEIN EXPRESSION; RNA SEQUENCE; WNT SIGNALING PATHWAY; CELL LINE; CYTOLOGY; GENE EXPRESSION; GENETIC TRANSCRIPTION; GENETICS; INDUCED PLURIPOTENT STEM CELL; METABOLISM; MUTATION; NERVE CELL; NEURAL STEM CELL; PHENOTYPE; TIME FACTOR;

BIOMARKERS; CELL DIFFERENTIATION; CELL LINE; FRONTOTEMPORAL DEMENTIA; GENE EXPRESSION; GENE EXPRESSION PROFILING; HAPLOINSUFFICIENCY; HUMANS; INDUCED PLURIPOTENT STEM CELLS; INTERCELLULAR SIGNALING PEPTIDES AND PROTEINS; MUTATION; NEURAL STEM CELLS; NEUROGENESIS; NEURONS; PHENOTYPE; TIME FACTORS; TRANSCRIPTION, GENETIC; TRANSCRIPTOME; WNT SIGNALING PATHWAY;

EID: 84920837939     PISSN: 22136711     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.stemcr.2014.12.001     Document Type: Article

References (27)

1. S. Almeida, Z. Zhang, G. Coppola, W. Mao, K. Futai, A. Karydas, M.D. Geschwind, M.C. Tartaglia, F. Gao, and D. Gianni Induced pluripotent stem cell models of progranulin-deficient frontotemporal dementia uncover specific reversible neuronal defects Cell Reports 2 2012 789 798
2. M. Baker, I.R. Mackenzie, S.M. Pickering-Brown, J. Gass, R. Rademakers, C. Lindholm, J. Snowden, J. Adamson, A.D. Sadovnick, and S. Rollinson Mutations in progranulin cause tau-negative frontotemporal dementia linked to chromosome 17 Nature 442 2006 916 919
3. M. Cruts, I. Gijselinck, J. van der Zee, S. Engelborghs, H. Wils, D. Pirici, R. Rademakers, R. Vandenberghe, B. Dermaut, and J.J. Martin Null mutations in progranulin cause ubiquitin-positive frontotemporal dementia linked to chromosome 17q21 Nature 442 2006 920 924
4. L. De Muynck, S. Herdewyn, S. Beel, W. Scheveneels, L. Van Den Bosch, W. Robberecht, and P. Van Damme The neurotrophic properties of progranulin depend on the granulin E domain but do not require sortilin binding Neurobiol. Aging 34 2013 2541 2547
5. M. DeJesus-Hernandez, I.R. Mackenzie, B.F. Boeve, A.L. Boxer, M. Baker, N.J. Rutherford, A.M. Nicholson, N.A. Finch, H. Flynn, and J. Adamson Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS Neuron 72 2011 245 256
6. I. Espuny-Camacho, K.A. Michelsen, D. Gall, D. Linaro, A. Hasche, J. Bonnefont, C. Bali, D. Orduz, A. Bilheu, and A. Herpoel Pyramidal neurons derived from human pluripotent stem cells integrate efficiently into mouse brain circuits in vivo Neuron 77 2013 440 456
7. X. Gao, A.P. Joselin, L. Wang, A. Kar, P. Ray, A. Bateman, A.M. Goate, and J.Y. Wu Progranulin promotes neurite outgrowth and neuronal differentiation by regulating GSK-3β Protein Cell. 1 2010 552 562
8. J. Gass, W.C. Lee, C. Cook, N. Finch, C. Stetler, K. Jansen-West, J. Lewis, C.D. Link, R. Rademakers, A. Nykjær, and L. Petrucelli Progranulin regulates neuronal outgrowth independent of sortilin Mol. Neurodegener. 7 2012 33
9. J.S. Goldman, J.M. Farmer, E.M. Wood, J.K. Johnson, A. Boxer, J. Neuhaus, C. Lomen-Hoerth, K.C. Wilhelmsen, V.M. Lee, M. Grossman, and B.L. Miller Comparison of family histories in FTLD subtypes and related tauopathies Neurology 65 2005 1817 1819
10. B.Y. Hu, and S.C. Zhang Differentiation of spinal motor neurons from pluripotent human stem cells Nat. Protoc. 4 2009 1295 1304
11. M. Hutton, C.L. Lendon, P. Rizzu, M. Baker, S. Froelich, H. Houlden, S. Pickering-Brown, S. Chakraverty, A. Isaacs, and A. Grover Association of missense and 5′-splice-site mutations in tau with the inherited dementia FTDP-17 Nature 393 1998 702 705
12. Y. Kayasuga, S. Chiba, M. Suzuki, T. Kikusui, T. Matsuwaki, K. Yamanouchi, H. Kotaki, R. Horai, Y. Iwakura, and M. Nishihara Alteration of behavioural phenotype in mice by targeted disruption of the progranulin gene Behav. Brain Res. 185 2007 110 118
13. C. Kim Disease modeling and cell based therapy with iPSC: future therapeutic option with fast and safe application Blood Res. 49 2014 7 14
14. Z. Korade, and K. Mirnics Wnt signaling as a potential therapeutic target for frontotemporal dementia Neuron 71 2011 955 957
15. H.L. Li, H.H. Wang, S.J. Liu, Y.Q. Deng, Y.J. Zhang, Q. Tian, X.C. Wang, X.Q. Chen, Y. Yang, and J.Y. Zhang Phosphorylation of tau antagonizes apoptosis by stabilizing beta-catenin, a mechanism involved in Alzheimer's neurodegeneration Proc. Natl. Acad. Sci. USA 104 2007 3591 3596
16. G.L. Ming, and H. Song Adult neurogenesis in the mammalian brain: significant answers and significant questions Neuron 70 2011 687 702
17. C.J. Miranda, L. Braun, Y. Jiang, M.E. Hester, L. Zhang, M. Riolo, H. Wang, M. Rao, R.A. Altura, and B.K. Kaspar Aging brain microenvironment decreases hippocampal neurogenesis through Wnt-mediated survivin signaling Aging Cell 11 2012 542 552
18. D. Neary, J. Snowden, and D. Mann Frontotemporal dementia Lancet Neurol. 4 2005 771 780
19. C.H. Ong, Z. He, L. Kriazhev, X. Shan, R.G. Palfree, and A. Bateman Regulation of progranulin expression in myeloid cells Am. J. Physiol. Regul. Integr. Comp. Physiol. 291 2006 R1602 R1612
20. L. Qiang, R. Fujita, and A. Abeliovich Remodeling neurodegeneration: somatic cell reprogramming-based models of adult neurological disorders Neuron 78 2013 957 969
21. A.E. Renton, E. Majounie, A. Waite, J. Simón-Sánchez, S. Rollinson, J.R. Gibbs, J.C. Schymick, H. Laaksovirta, J.C. van Swieten, L. Myllykangas ITALSGEN Consortium A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD Neuron 72 2011 257 268
22. E.Y. Rosen, E.M. Wexler, R. Versano, G. Coppola, F. Gao, K.D. Winden, M.C. Oldham, L.H. Martens, P. Zhou, R.V. Farese Jr., and D.H. Geschwind Functional genomic analyses identify pathways dysregulated by progranulin deficiency, implicating Wnt signaling Neuron 71 2011 1030 1042
23. K. Takahashi, K. Okita, M. Nakagawa, and S. Yamanaka Induction of pluripotent stem cells from fibroblast cultures Nat. Protoc. 2 2007 3081 3089
24. 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
25. P. Van Damme, A. Van Hoecke, D. Lambrechts, P. Vanacker, E. Bogaert, J. van Swieten, P. Carmeliet, L. Van Den Bosch, and W. Robberecht Progranulin functions as a neurotrophic factor to regulate neurite outgrowth and enhance neuronal survival J. Cell Biol. 181 2008 37 41
26. J.C. van Swieten, and P. Heutink Mutations in progranulin (GRN) within the spectrum of clinical and pathological phenotypes of frontotemporal dementia Lancet Neurol. 7 2008 965 974
27. E.M. Wexler, A. Paucer, H.I. Kornblum, T.D. Palmer, and D.H. Geschwind Endogenous Wnt signaling maintains neural progenitor cell potency Stem Cells 27 2009 1130 1141