Use of human pluripotent stem cell-derived cells for neurodegenerative disease modeling and drug screening platform

被引:22
作者
Antonio Garcia-Leon, Juan [1 ,2 ]
Vitorica, Javier [2 ,3 ,4 ]
Gutierrez, Antonia [1 ,2 ]
机构
[1] Univ Malaga, Dept Biol Celular Genet & Fisiol, Fac Ciencias, Inst Invest Biomed Malaga IBIMA, Malaga 29010, Spain
[2] Ctr Invest Biomed Red Enfermedades Neurodegenerat, Madrid 28031, Spain
[3] Univ Seville, Fac Farm, Dept Bioquim & Biol Mol, E-41012 Seville, Spain
[4] Univ Seville, CSIC, Hosp Univ Virgen del Rocio, Inst Biomed Sevilla IBiS, Seville 41013, Spain
关键词
Alzheimer's disease; amyotrophic lateral sclerosis; CRISPR-Cas9; disease modeling; drug screening; hiPSCs; human-induced pluripotent stem cells; Huntington's disease; multiple sclerosis; neurodegenerative disease; Parkinson's disease; MICROGLIA-LIKE CELLS; OLIGODENDROCYTE PROGENITOR CELLS; FAMILIAL ALZHEIMERS-DISEASE; DOUBLE-STRAND BREAKS; IN-VITRO; EFFICIENT GENERATION; CEREBRAL ORGANOIDS; MOTOR-NEURONS; MOUSE MODEL; DIFFERENTIATION;
D O I
10.4155/fmc-2018-0520
中图分类号
R914 [药物化学];
学科分类号
100701 ;
摘要
Most neurodegenerative diseases are characterized by a complex and mostly still unresolved pathology. This fact, together with the lack of reliable disease models, has precluded the development of effective therapies counteracting the disease progression. The advent of human pluripotent stem cells has revolutionized the field allowing the generation of disease-relevant neural cell types that can be used for disease modeling, drug screening and, possibly, cell transplantation purposes. In this Review, we discuss the applications of human pluripotent stem cells, the development of efficient protocols for the derivation of the different neural cells and their applicability for robust in vitro disease modeling and drug screening platforms for most common neurodegenerative conditions.
引用
收藏
页码:1305 / 1322
页数:18
相关论文
共 154 条
[1]   iPSC-Derived Human Microglia-like Cells to Study Neurological Diseases [J].
Abud, Edsel M. ;
Ramirez, Ricardo N. ;
Martinez, Eric S. ;
Healy, Luke M. ;
Nguyen, Cecilia H. H. ;
Newman, Sean A. ;
Yeromin, Andriy V. ;
Scarfone, Vanessa M. ;
Marsh, Samuel E. ;
Fimbres, Cristhian ;
Caraway, Chad A. ;
Fote, Gianna M. ;
Madany, Abdullah M. ;
Agrawal, Anshu ;
Kayed, Rakez ;
Gylys, Karen H. ;
Cahalan, Michael D. ;
Cummings, Brian J. ;
Antel, Jack P. ;
Mortazavi, Ali ;
Carson, Monica J. ;
Poon, Wayne W. ;
Blurton-Jones, Mathew .
NEURON, 2017, 94 (02) :278-+
[2]   In vivo CRISPR editing with no detectable genome-wide off-target mutations [J].
Akcakaya, Pinar ;
Bobbin, Maggie L. ;
Guo, Jimmy A. ;
Malagon-Lopez, Jose ;
Clement, Kendell ;
Garcia, Sara P. ;
Fellows, Mick D. ;
Porritt, Michelle J. ;
Firth, Mike A. ;
Carreras, Alba ;
Baccega, Tania ;
Seeliger, Frank ;
Bjursell, Mikael ;
Tsai, Shengdar Q. ;
Nguyen, Nhu T. ;
Nitsch, Roberto ;
Mayr, Lorenz M. ;
Pinello, Luca ;
Bohlool-Y, Mohammad ;
Aryee, Martin J. ;
Maresca, Marcello ;
Joung, J. Keith .
NATURE, 2018, 561 (7723) :416-+
[3]   Accelerated High-Yield Generation of Limb-Innervating Motor Neurons from Human Stem Cells [J].
Amoroso, Mackenzie W. ;
Croft, Gist F. ;
Williams, Damian J. ;
O'Keeffe, Sean ;
Carrasco, Monica A. ;
Davis, Anne R. ;
Roybon, Laurent ;
Oakley, Derek H. ;
Maniatis, Tom ;
Henderson, Christopher E. ;
Wichterle, Hynek .
JOURNAL OF NEUROSCIENCE, 2013, 33 (02) :574-586
[4]   CRISPR off-targets: a reassessment [J].
不详 .
NATURE METHODS, 2018, 15 (04) :229-230
[5]  
[Anonymous], 2015, BRAIN 11, DOI DOI 10.1093/BRAIN/AWV222
[6]   Projected increase in amyotrophic lateral sclerosis from 2015 to 2040 [J].
Arthur, Karissa C. ;
Calvo, Andrea ;
Price, T. Ryan ;
Geiger, Joshua T. ;
Chio, Adriano ;
Traynor, Bryan J. .
NATURE COMMUNICATIONS, 2016, 7
[7]   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
[8]   Huntington disease [J].
Bates, Gillian P. ;
Dorsey, Ray ;
Gusella, James F. ;
Hayden, Michael R. ;
Kay, Chris ;
Leavitt, Blair R. ;
Nance, Martha ;
Ross, Christopher A. ;
Scahill, Rachael I. ;
Wetzel, Ronald ;
Wild, Edward J. ;
Tabrizi, Sarah J. .
NATURE REVIEWS DISEASE PRIMERS, 2015, 1
[9]   Intrinsic and adaptive myelinationA sequential mechanism for smart wiring in the brain [J].
Bechler, Marie E. ;
Swire, Matthew ;
Ffrench-Constant, Charles .
DEVELOPMENTAL NEUROBIOLOGY, 2018, 78 (02) :68-79
[10]   Are the Prevalence and Incidence of Multiple Sclerosis Changing? [J].
Benito-Leon, Julian .
NEUROEPIDEMIOLOGY, 2011, 36 (03) :148-149