Pluripotent stem cells in disease modelling and drug discovery

被引：431

作者：

Avior, Yishai ^{[1
]}

Sagi, Ido ^{[1
]}

Benvenisty, Nissim ^{[1
]}

机构：

[1] Hebrew Univ Jerusalem, Inst Life Sci, Azrieli Ctr Stem Cells & Genet Res, IL-91904 Jerusalem, Israel

来源：

NATURE REVIEWS MOLECULAR CELL BIOLOGY | 2016年 / 17卷 / 03期

基金：

以色列科学基金会;

关键词：

FRAGILE-X-SYNDROME; FAMILIAL ALZHEIMERS-DISEASE; MOTOR-NEURONS; CELLULAR PHENOTYPES; TARGETED DISRUPTION; NEURAL DERIVATIVES; PARKINSONS-DISEASE; GENE-EXPRESSION; PATIENT; ALS;

D O I：

10.1038/nrm.2015.27

中图分类号：

Q2 [细胞生物学];

学科分类号：

071013 [干细胞生物学];

摘要：

Experimental modelling of human disorders enables the definition of the cellular and molecular mechanisms underlying diseases and the development of therapies for treating them. The availability of human pluripotent stem cells (PSCs), which are capable of self-renewal and have the potential to differentiate into virtually any cell type, can now help to overcome the limitations of animal models for certain disorders. The ability to model human diseases using cultured PSCs has revolutionized the ways in which we study monogenic, complex and epigenetic disorders, as well as early- and late-onset diseases. Several strategies are used to generate such disease models using either embryonic stem cells (ES cells) or patient-specific induced PSCs (iPSCs), creating new possibilities for the establishment of models and their use in drug screening.

引用

页码：170 / 182

页数：13

共 150 条

[1]

Characterization of human embryonic stem cell lines by the International Stem Cell Initiative [J].

Adewumi, Oluseun ;

Aflatoonian, Behrouz ;

Ahrlund-Richter, Lars ;

Amit, Michal ;

Andrews, Peter W. ;

Beighton, Gemma ;

Bello, Paul A. ;

Benvenisty, Nissim ;

Berry, Lorraine S. ;

Bevan, Simon ;

Blum, Barak ;

Brooking, Justin ;

Chen, Kevin G. ;

Choo, Andre B. H. ;

Churchill, Gary A. ;

Corbel, Marie ;

Damjanov, Ivan ;

Draper, Jon S. ;

Dvorak, Petr ;

Emanuelsson, Katarina ;

Fleck, Roland A. ;

Ford, Angela ;

Gertow, Karin ;

Gertsenstein, Marina ;

Gokhale, Paul J. ;

Hamilton, Rebecca S. ;

Hampl, Ales ;

Healy, Lyn E. ;

Hovatta, Outi ;

Hyllner, Johan ;

Imreh, Marta P. ;

Itskovitz-Eldor, Joseph ;

Jackson, Jamie ;

Johnson, Jacqueline L. ;

Jones, Mark ;

Kee, Kehkooi ;

King, Benjamin L. ;

Knowles, Barbara B. ;

Lako, Majlinda ;

Lebrin, Franck ;

Mallon, Barbara S. ;

Manning, Daisy ;

Mayshar, Yoav ;

Mckay, Ronald D. G. ;

Michalska, Anna E. ;

Mikkola, Milla ;

Mileikovsky, Masha ;

Minger, Stephen L. ;

Moore, Harry D. ;

Mummery, Christine L. .

NATURE BIOTECHNOLOGY, 2007, 25 (07) :803-816

[2]

Molecular analysis of FMR1 reactivation in fragile-X induced pluripotent stem cells and their neuronal derivatives [J].

Bar-Nur, Ori ;

Caspi, Inbal ;

Benvenisty, Nissim .

JOURNAL OF MOLECULAR CELL BIOLOGY, 2012, 4 (03) :180-183

[3]

Epigenetic Memory and Preferential Lineage-Specific Differentiation in Induced Pluripotent Stem Cells Derived from Human Pancreatic Islet Beta Cells [J].

Bar-Nur, Ori ;

Russ, Holger A. ;

Efrat, Shimon ;

Benvenisty, Nissim .

CELL STEM CELL, 2011, 9 (01) :17-23

[4]

Autophagy induction enhances TDP43 turnover and survival in neuronal ALS models [J].

Barmada, Sami J. ;

Serio, Andrea ;

Arjun, Arpana ;

Bilican, Bilada ;

Daub, Aaron ;

Ando, D. Michael ;

Tsvetkov, Andrey ;

Pleiss, Michael ;

Li, Xingli ;

Peisach, Daniel ;

Shaw, Christopher ;

Chandran, Siddharthan ;

Finkbeiner, Steven .

NATURE CHEMICAL BIOLOGY, 2014, 10 (08) :677-U119

[5]

Induced pluripotent stem cells: the new patient? [J].

Bellin, Milena ;

Marchetto, Maria C. ;

Gage, Fred H. ;

Mummery, Christine L. .

NATURE REVIEWS MOLECULAR CELL BIOLOGY, 2012, 13 (11) :713-726

[6]

Human Embryonic Stem Cells as Models for Aneuploid Chromosomal Syndromes [J].

Biancotti, Juan-Carlos ;

Narwani, Kavita ;

Buehler, Nicole ;

Mandefro, Berhan ;

Golan-Lev, Tamar ;

Yanuka, Ofra ;

Clark, Amander ;

Hill, David ;

Benvenisty, Nissim ;

Lavon, Neta .

STEM CELLS, 2010, 28 (09) :1530-1540

[7]

Modelling schizophrenia using human induced pluripotent stem cells [J].

Brennand, Kristen J. ;

Simone, Anthony ;

Jou, Jessica ;

Gelboin-Burkhart, Chelsea ;

Tran, Ngoc ;

Sangar, Sarah ;

Li, Yan ;

Mu, Yangling ;

Chen, Gong ;

Yu, Diana ;

McCarthy, Shane ;

Sebat, Jonathan ;

Gage, Fred H. .

NATURE, 2011, 473 (7346) :221-+

[8]

Cell Lines Derived from Human Parthenogenetic Embryos Can Display Aberrant Centriole Distribution and Altered Expression Levels of Mitotic Spindle Check-point Transcripts [J].

Brevini, Tiziana A. L. ;

Pennarossa, Georgia ;

Antonini, Stefania ;

Paffoni, Alessio ;

Tettamanti, Gianluca ;

Montemurro, Tiziana ;

Radaelli, Enrico ;

Lazzari, Lorenza ;

Rebulla, Paolo ;

Scanziani, Eugenio ;

de Eguileor, Magda ;

Benvenisty, Nissim ;

Ragni, Guido ;

Gandolfi, Fulvio .

STEM CELL REVIEWS AND REPORTS, 2009, 5 (04) :340-352

[9]

Integration-Free Induced Pluripotent Stem Cells Model Genetic and Neural Developmental Features of Down Syndrome Etiology [J].

Briggs, James A. ;

Sun, Jane ;

Shepherd, Jill ;

Ovchinnikov, Dmitry A. ;

Chung, Tung-Liang ;

Nayler, Sam P. ;

Kao, Li-Pin ;

Morrow, Carl A. ;

Thakar, Nilay Y. ;

Soo, Set-Yen ;

Peura, Teija ;

Grimmond, Sean ;

Wolvetang, Ernst J. .

STEM CELLS, 2013, 31 (03) :467-478

[10]

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

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