Human iPSC application in Alzheimer's disease and Tau-related neurodegenerative diseases

被引:27
作者
Julia, T. C. W. [1 ]
机构
[1] Icahn Sch Med Mt Sinai, Dept Neurosci, Ronald M Loeb Ctr Alzheimers Dis, New York, NY 10029 USA
关键词
Alzheimer's disease (AD); Induced pluripotent stem cells (iPSCs); Disease modeling; Tauophaty; PLURIPOTENT STEM-CELLS; FUNCTIONAL CORTICAL-NEURONS; GAMMA-SECRETASE ACTIVITY; FRONTOTEMPORAL DEMENTIA; APOLIPOPROTEIN E4; AMYLOID-BETA; DIFFERENTIATION; MICROGLIA; MODEL; MUTATION;
D O I
10.1016/j.neulet.2019.01.043
中图分类号
Q189 [神经科学];
学科分类号
071006 ;
摘要
Alzheimer's disease (AD) is a chronic neurodegenerative disorder characterized by beta-amyloid (A beta) plaques and neurofibrillary tangles with progressive cognitive decline. After years of research with failed clinical trials surrounding A beta and tau using numerous in vitro and in vivo AD transgenic animals, human model is necessary to advance our understanding of AD and drug discovery. Induced pluripotent stem cells (iPSCs) offers a novel approach for the establishment of a human model that reflects human genetics and physiology to investigate mechanisms of AD and other forms of dementia, which potentially could lead to a drug discovery, followed by treatment for the disease. In this review, I discuss the advantages in using iPSC technology with various forms of modeling, the progress that has been made to date in patient-derived AD iPSC model and challenges posed by using this technology. Finally, I suggest the future directions of disease modeling and the potential of iPSC technology in AD and other neurodegenerative research.
引用
收藏
页码:31 / 40
页数:10
相关论文
共 99 条
[1]  
Abud E.M., IPSC DERIVED HUMAN M
[2]   Neuronal medium that supports basic synaptic functions and activity of human neurons in vitro [J].
Bardy, Cedric ;
van den Hurk, Mark ;
Eames, Tameji ;
Marchand, Cynthia ;
Hernandez, Ruben V. ;
Kellogg, Mariko ;
Gorris, Mark ;
Galet, Ben ;
Palomares, Vanessa ;
Brown, Joshua ;
Bang, Anne G. ;
Mertens, Jerome ;
Boehnke, Lena ;
Boyer, Leah ;
Simon, Suzanne ;
Gage, Fred H. .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2015, 112 (20) :E2725-E2734
[3]   Contribution to Alzheimer's disease risk of rare variants in TREM2, SORL1, and ABCA7 in 1779 cases and 1273 controls [J].
Bellenguez, Celine ;
Charbonnier, Camille ;
Grenier-Boley, Benjamin ;
Quenez, Olivier ;
Le Guennec, Kilan ;
Nicolas, Gael ;
Chauhan, Ganesh ;
Wallon, David ;
Rousseau, Stephane ;
Richard, Anne Claire ;
Boland, Anne ;
Bourque, Guillaume ;
Munter, Hans Markus ;
Olaso, Robert ;
Meyer, Vincent ;
Rollin-Sillaire, Adeline ;
Pasquier, Florence ;
Letenneur, Luc ;
Redon, Richard ;
Dartigues, Jean-Francois ;
Tzourio, Christophe ;
Frebourg, Thierry ;
Lathrop, Mark ;
Deleuze, Jean-Francois ;
Hannequin, Didier ;
Genin, Emmanuelle ;
Amouyel, Philippe ;
Debette, Stephanie ;
Lambert, Jean-Charles ;
Campion, Dominique .
NEUROBIOLOGY OF AGING, 2017, 59 :220.e1-220.e9
[4]  
Biswas M.H.U., 2016, STEM CELL REP, DOI [10.1016/j.stemer.2016.08.006, DOI 10.1016/J.STEMER.2016.08.006]
[5]   Structure-dependent Impairment of Intracellular Apolipoprotein E4 Trafficking and Its Detrimental Effects Are Rescued by Small-molecule Structure Correctors [J].
Brodbeck, Jens ;
McGuire, Jim ;
Liu, Zhaoping ;
Meyer-Franke, Anke ;
Balestra, Maureen E. ;
Jeong, Dah-eun ;
Pleiss, Mike ;
McComas, Casey ;
Hess, Fred ;
Witter, David ;
Peterson, Scott ;
Childers, Matthew ;
Goulet, Mark ;
Liverton, Nigel ;
Hargreaves, Richard ;
Freedman, Stephen ;
Weisgraber, Karl H. ;
Mahley, Robert W. ;
Huang, Yadong .
JOURNAL OF BIOLOGICAL CHEMISTRY, 2011, 286 (19) :17217-17226
[6]   Origins and implications of pluripotent stem cell variability and heterogeneity [J].
Cahan, Patrick ;
Daley, George Q. .
NATURE REVIEWS MOLECULAR CELL BIOLOGY, 2013, 14 (06) :357-368
[7]   Human cerebral organoids recapitulate gene expression programs of fetal neocortex development [J].
Camp, J. Gray ;
Badsha, Farhath ;
Florio, Marta ;
Kanton, Sabina ;
Gerber, Tobias ;
Wilsch-Braeuninger, Michaela ;
Lewitus, Eric ;
Sykes, Alex ;
Hevers, Wulf ;
Lancaster, Madeline ;
Knoblich, Juergen A. ;
Lachmann, Robert ;
Paeaebo, Svante ;
Huttner, Wieland B. ;
Treutlein, Barbara .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2015, 112 (51) :15672-15677
[8]   Small Molecule Structure Correctors Abolish Detrimental Effects of Apolipoprotein E4 in Cultured Neurons [J].
Chen, Hung-Kai ;
Liu, Zhaoping ;
Meyer-Franke, Anke ;
Brodbeck, Jens ;
Miranda, Rene D. ;
McGuire, James G. ;
Pleiss, Michael A. ;
Ji, Zhong-Sheng ;
Balestra, Maureen E. ;
Walker, David W. ;
Xu, Qin ;
Jeong, Dah-eun ;
Budamagunta, Madhu S. ;
Voss, John C. ;
Freedman, Stephen B. ;
Weisgraber, Karl H. ;
Huang, Yadong ;
Mahley, Robert W. .
JOURNAL OF BIOLOGICAL CHEMISTRY, 2012, 287 (08) :5253-5266
[9]   Multiplex Genome Engineering Using CRISPR/Cas Systems [J].
Cong, Le ;
Ran, F. Ann ;
Cox, David ;
Lin, Shuailiang ;
Barretto, Robert ;
Habib, Naomi ;
Hsu, Patrick D. ;
Wu, Xuebing ;
Jiang, Wenyan ;
Marraffini, Luciano A. ;
Zhang, Feng .
SCIENCE, 2013, 339 (6121) :819-823
[10]   Quantitative analysis of tau isoform transcripts in sporadic tauopathies [J].
Connell, JW ;
Rodriguez-Martin, T ;
Gibb, GM ;
Kahn, NM ;
Grierson, AJ ;
Hanger, DP ;
Revesz, T ;
Lantos, PL ;
Anderton, BH ;
Gallo, JM .
MOLECULAR BRAIN RESEARCH, 2005, 137 (1-2) :104-109