Les cellules neuronales dérivées des cellules souches pluripotentes induites humaines : modélisation des maladies du motoneurone

作者: Delphine Bohl

DOI: 10.1051/JBIO/2016004

关键词:

摘要: Among motor neuron diseases, spinal muscular atrophy type 1 and amyotrophic lateral sclerosis are very aggressive diseases with no cure. With the breakthrough of human induced pluripotent stem cells, iPS, researchers have now at their disposal a powerful tool to generate neurons in culture study pathological defects patient cells. In this review, we will see which tools for patients motoneurons were developed from iPS cells different cellular models that generated. We also how these validated current research identify new therapeutic leads.

参考文章(43)
Diana Toli, Dorothée Buttigieg, Stéphane Blanchard, Thomas Lemonnier, Boris Lamotte d’Incamps, Sarah Bellouze, Gilbert Baillat, Delphine Bohl, Georg Haase, Modeling amyotrophic lateral sclerosis in pure human iPSc-derived motor neurons isolated by a novel FACS double selection technique Neurobiology of Disease. ,vol. 82, pp. 269- 280 ,(2015) , 10.1016/J.NBD.2015.06.011
María G Boza-Morán, Rebeca Martínez-Hernández, Sara Bernal, Klaus Wanisch, Eva Also-Rallo, Anita Le Heron, Laura Alías, Cécile Denis, Mathilde Girard, Jiing-Kuan Yee, Eduardo F. Tizzano, Rafael J Yáñez-Muñoz, Decay in survival motor neuron and plastin 3 levels during differentiation of iPSC-derived human motor neurons. Scientific Reports. ,vol. 5, pp. 11696- 11696 ,(2015) , 10.1038/SREP11696
Julia Japtok, Xenia Lojewski, Marcel Naumann, Moritz Klingenstein, Peter Reinhardt, Jared Sterneckert, Stefan Putz, Maria Demestre, Tobias M. Boeckers, Albert C. Ludolph, Stefan Liebau, Alexander Storch, Andreas Hermann, Stepwise acquirement of hallmark neuropathology in FUS-ALS iPSC models depends on mutation type and neuronal aging. Neurobiology of Disease. ,vol. 82, pp. 420- 429 ,(2015) , 10.1016/J.NBD.2015.07.017
Xiaowei W. Su, James R. Broach, James R. Connor, Glenn S. Gerhard, Zachary Simmons, Genetic heterogeneity of amyotrophic lateral sclerosis: Implications for clinical practice and research Muscle & Nerve. ,vol. 49, pp. 786- 803 ,(2014) , 10.1002/MUS.24198
Jacqueline T. Pham, Aaron R. Haeusler, Nipun A. Mistry, Svetlana Vidensky, Elizabeth L. Daley, Erin M. Poth, Benjamin Hoover, Daniel M. Fines, Nicholas Maragakis, Pentti J. Tienari, Leonard Petrucelli, Bryan J. Traynor, Jiou Wang, Frank Rigo, C. Frank Bennett, Seth Blackshaw, Rita Sattler, Jeffrey D. Rothstein, Christopher J. Donnelly, Ping-Wu Zhang, RNA Toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention [Neuron, 79, (September 18, 2013), 1222-1231] Neuron. ,vol. 80, ,(2013) , 10.1016/J.NEURON.2013.10.055
Shi-Yan Ng, Boon Seng Soh, Natalia Rodriguez-Muela, David G. Hendrickson, Feodor Price, John L. Rinn, Lee L. Rubin, Genome-wide RNA-Seq of Human Motor Neurons Implicates Selective ER Stress Activation in Spinal Muscular Atrophy Cell Stem Cell. ,vol. 17, pp. 569- 584 ,(2015) , 10.1016/J.STEM.2015.08.003
Matthew F. Burkhardt, Fernando J. Martinez, Sarah Wright, Carla Ramos, Dmitri Volfson, Michael Mason, Jeff Garnes, Vu Dang, Jeffery Lievers, Uzma Shoukat-Mumtaz, Rita Martinez, Hui Gai, Robert Blake, Eugeni Vaisberg, Marica Grskovic, Charles Johnson, Stefan Irion, Jessica Bright, Bonnie Cooper, Leane Nguyen, Irene Griswold-Prenner, Ashkan Javaherian, A cellular model for sporadic ALS using patient-derived induced pluripotent stem cells Molecular and Cellular Neuroscience. ,vol. 56, pp. 355- 364 ,(2013) , 10.1016/J.MCN.2013.07.007
Agnes L. Nishimura, Carole Shum, Emma L. Scotter, Amr Abdelgany, Valentina Sardone, Jamie Wright, Youn-Bok Lee, Han-Jou Chen, Bilada Bilican, Monica Carrasco, Tom Maniatis, Siddharthan Chandran, Boris Rogelj, Jean-Marc Gallo, Christopher E. Shaw, Allele-specific knockdown of ALS-associated mutant TDP-43 in neural stem cells derived from induced pluripotent stem cells PLOS ONE. ,vol. 9, ,(2014) , 10.1371/JOURNAL.PONE.0091269
Yves Maury, Julien Côme, Rebecca A Piskorowski, Nouzha Salah-Mohellibi, Vivien Chevaleyre, Marc Peschanski, Cécile Martinat, Stéphane Nedelec, Combinatorial analysis of developmental cues efficiently converts human pluripotent stem cells into multiple neuronal subtypes Nature Biotechnology. ,vol. 33, pp. 89- 96 ,(2015) , 10.1038/NBT.3049
A. Serio, B. Bilican, S. J. Barmada, D. M. Ando, C. Zhao, R. Siller, K. Burr, G. Haghi, D. Story, A. L. Nishimura, M. A. Carrasco, H. P. Phatnani, C. Shum, I. Wilmut, T. Maniatis, C. E. Shaw, S. Finkbeiner, S. Chandran, Astrocyte pathology and the absence of non-cell autonomy in an induced pluripotent stem cell model of TDP-43 proteinopathy Proceedings of the National Academy of Sciences of the United States of America. ,vol. 110, pp. 4697- 4702 ,(2013) , 10.1073/PNAS.1300398110