Induced Pluripotent Stem Cells A Powerful Neurodegenerative Disease Modeling Tool for Mechanism Study and Drug Discovery

被引:19
作者
Chang, Chia-Yu [1 ,2 ]
Ting, Hsiao-Chien [1 ]
Liu, Ching-Ann [1 ,2 ]
Su, Hong-Lin [3 ]
Chiou, Tzyy-Wen [4 ]
Harn, Horng-Jyh [1 ,5 ,6 ]
Lin, Shinn-Zong [1 ,7 ]
机构
[1] Buddhist Tzu Chi Med Fdn, Bioinnovat Ctr, Hualien, Taiwan
[2] Buddhist Tzu Chi Gen Hosp, Dept Med Res, Hualien, Taiwan
[3] Natl Chung Hsing Univ, Dept Life Sci, Taichung, Taiwan
[4] Natl Dong Hwa Univ, Dept Life Sci, Hualien, Taiwan
[5] Buddhist Tzu Chi Gen Hosp, Dept Pathol, Hualien, Taiwan
[6] Tzu Chi Univ, Hualien, Taiwan
[7] Buddhist Tzu Chi Gen Hosp, Dept Neurosurg, Hualien, Taiwan
关键词
Neurodegenerative disease; induced pluripotent stem cells (iPSCs); disease modeling; neuronal differentiation; mechanism study; drug screening; SPINAL MUSCULAR-ATROPHY; FAMILIAL ALZHEIMERS-DISEASE; MIDBRAIN DOPAMINE NEURONS; MOTOR-NEURONS; HUMAN ES; DIRECTED DIFFERENTIATION; CEREBRAL ORGANOIDS; GENETIC CORRECTION; SELF-ORGANIZATION; IPSCS REVEALS;
D O I
10.1177/0963689718775406
中图分类号
Q813 [细胞工程];
学科分类号
摘要
Many neurodegenerative diseases are progressive, complex diseases without clear mechanisms or effective treatments. To study the mechanisms underlying these diseases and to develop treatment strategies, a reliable in vitro modeling system is critical. Induced pluripotent stem cells (iPSCs) have the ability to self-renew and possess the differentiation potential to become any kind of adult cell; thus, they may serve as a powerful material for disease modeling. Indeed, patient cell-derived iPSCs can differentiate into specific cell lineages that display the appropriate disease phenotypes and vulnerabilities. In this review, we highlight neuronal differentiation methods and the current development of iPSC-based neurodegenerative disease modeling tools for mechanism study and drug screening, with a discussion of the challenges and future inspiration for application.
引用
收藏
页码:1588 / 1602
页数:15
相关论文
共 115 条
[1]  
Alves CJ, 2015, FRONT CELL NEUROSCI, V9, DOI [10.3389/fncel.2015.00332, 10.3389/fncel.2015.00289]
[2]   Genetic Correction of Huntington's Disease Phenotypes in Induced Pluripotent Stem Cells [J].
An, Mahru C. ;
Zhang, Ningzhe ;
Scott, Gary ;
Montoro, Daniel ;
Wittkop, Tobias ;
Mooney, Sean ;
Melov, Simon ;
Ellerby, Lisa M. .
CELL STEM CELL, 2012, 11 (02) :253-263
[3]   The selectivity of protein kinase inhibitors: a further update [J].
Bain, Jenny ;
Plater, Lorna ;
Elliott, Matt ;
Shpiro, Natalia ;
Hastie, C. James ;
Mclauchlan, Hilary ;
Klevernic, Iva ;
Arthur, J. Simon C. ;
Alessi, Dario R. ;
Cohen, Philip .
BIOCHEMICAL JOURNAL, 2007, 408 :297-315
[4]   Passaging and colony expansion of human pluripotent stem cells by enzyme-free dissociation in chemically defined culture conditions [J].
Beers, Jeanette ;
Gulbranson, Daniel R. ;
George, Nicole ;
Siniscalchi, Lauren I. ;
Jones, Jeffrey ;
Thomson, James A. ;
Chen, Guokai .
NATURE PROTOCOLS, 2012, 7 (11) :2029-2040
[5]   Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors [J].
Boch, Jens ;
Scholze, Heidi ;
Schornack, Sebastian ;
Landgraf, Angelika ;
Hahn, Simone ;
Kay, Sabine ;
Lahaye, Thomas ;
Nickstadt, Anja ;
Bonas, Ulla .
SCIENCE, 2009, 326 (5959) :1509-1512
[6]   TAL Effectors: Customizable Proteins for DNA Targeting [J].
Bogdanove, Adam J. ;
Voytas, Daniel F. .
SCIENCE, 2011, 333 (6051) :1843-1846
[7]   A cellular model for sporadic ALS using patient-derived induced pluripotent stem cells [J].
Burkhardt, Matthew F. ;
Martinez, Fernando J. ;
Wright, Sarah ;
Ramos, Carla ;
Volfson, Dmitri ;
Mason, Michael ;
Garnes, Jeff ;
Vu Dang ;
Lievers, Jeffery ;
Shoukat-Mumtaz, Uzma ;
Martinez, Rita ;
Gai, Hui ;
Blake, Robert ;
Vaisberg, Eugeni ;
Grskovic, Marica ;
Johnson, Charles ;
Irion, Stefan ;
Bright, Jessica ;
Cooper, Bonnie ;
Leane Nguyen ;
Griswold-Prenner, Irene ;
Javaherian, Ashkan .
MOLECULAR AND CELLULAR NEUROSCIENCE, 2013, 56 :355-364
[8]  
CAIRESJUNIOR LC, 2018, NAT COMM, V0009
[9]   Genomic mapping of phosphorothioates reveals partial modification of short consensus sequences [J].
Cao, Bo ;
Chen, Chao ;
DeMott, Michael S. ;
Cheng, Qiuxiang ;
Clark, Tyson A. ;
Xiong, Xiaolin ;
Zheng, Xiaoqing ;
Butty, Vincent ;
Levine, Stuart S. ;
Yuan, George ;
Boitano, Matthew ;
Khai Luong ;
Song, Yi ;
Zhou, Xiufen ;
Deng, Zixin ;
Turner, Stephen W. ;
Korlach, Jonas ;
You, Delin ;
Wang, Lianrong ;
Chen, Shi ;
Dedon, Peter C. .
NATURE COMMUNICATIONS, 2014, 5
[10]   Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling [J].
Chambers, Stuart M. ;
Fasano, Christopher A. ;
Papapetrou, Eirini P. ;
Tomishima, Mark ;
Sadelain, Michel ;
Studer, Lorenz .
NATURE BIOTECHNOLOGY, 2009, 27 (03) :275-280