From Dish to Bedside: Lessons Learned While Translating Findings from a Stem Cell Model of Disease to a Clinical Trial
作者： John McNeish , Jason P. Gardner , Brian J. Wainger , Clifford J. Woolf , Kevin Eggan
DOI: 10.1016/J.STEM.2015.06.013
关键词: Disease 、 Clinical trial 、 Biology 、 Stem cell 、 Medical physics
摘要: … The first important lesson we learned from our desire to translate these studies was that the use of gene editing to correct the SOD1 mutation, and with it the physiological changes we …
core.ac.uk
elsevier.com
sci-hub.se 参考文章(9)
Mark C. Bellingham, A Review of the Neural Mechanisms of Action and Clinical Efficiency of Riluzole in Treating Amyotrophic Lateral Sclerosis: What have we Learned in the Last Decade? CNS Neuroscience & Therapeutics. ,vol. 17, pp. 4- 31 ,(2011) , 10.1111/J.1755-5949.2009.00116.X
Jong Seok Bae, Neil G. Simon, Parvathi Menon, Steve Vucic, Matthew C. Kiernan, The Puzzling Case of Hyperexcitability in Amyotrophic Lateral Sclerosis Journal of Clinical Neurology. ,vol. 9, pp. 65- 74 ,(2013) , 10.3988/JCN.2013.9.2.65
Steve Perrin, Preclinical research: Make mouse studies work Nature. ,vol. 507, pp. 423- 425 ,(2014) , 10.1038/507423A
Krishanu Saha, Linda F. Hogle, Allying with donors to link health and medical information with stem cell lines can advance disease modeling while enhancing data access. Cell Stem Cell. ,vol. 14, pp. 559- 560 ,(2014) , 10.1016/J.STEM.2014.04.012
John McNeish, Embryonic stem cells in drug discovery Nature Reviews Drug Discovery. ,vol. 3, pp. 70- 80 ,(2004) , 10.1038/NRD1281
Marica Grskovic, Ashkan Javaherian, Berta Strulovici, George Q. Daley, Induced pluripotent stem cells — opportunities for disease modelling and drug discovery Nature Reviews Drug Discovery. ,vol. 10, pp. 915- 929 ,(2011) , 10.1038/NRD3577
Evangelos Kiskinis, Jackson Sandoe, Luis A. Williams, Gabriella L. Boulting, Rob Moccia, Brian J. Wainger, Steve Han, Theodore Peng, Sebastian Thams, Shravani Mikkilineni, Cassidy Mellin, Florian T. Merkle, Brandi N. Davis-Dusenbery, Michael Ziller, Derek Oakley, Justin Ichida, Stefania Di Costanzo, Nick Atwater, Morgan L. Maeder, Mathew J. Goodwin, James Nemesh, Robert E. Handsaker, Daniel Paull, Scott Noggle, Steven A. McCarroll, J. Keith Joung, Clifford J. Woolf, Robert H. Brown, Kevin Eggan, Pathways disrupted in human ALS motor neurons identified through genetic correction of mutant SOD1. Cell Stem Cell. ,vol. 14, pp. 781- 795 ,(2014) , 10.1016/J.STEM.2014.03.004
Milena Bellin, Maria C Marchetto, Fred H Gage, Christine L Mummery, None, Induced pluripotent stem cells: the new patient? Nature Reviews Molecular Cell Biology. ,vol. 13, pp. 713- 726 ,(2012) , 10.1038/NRM3448
Brian J. Wainger, Evangelos Kiskinis, Cassidy Mellin, Ole Wiskow, Steve S.W. Han, Jackson Sandoe, Numa P. Perez, Luis A. Williams, Seungkyu Lee, Gabriella Boulting, James D. Berry, Robert H. Brown, Merit E. Cudkowicz, Bruce P. Bean, Kevin Eggan, Clifford J. Woolf, Intrinsic Membrane Hyperexcitability of Amyotrophic Lateral Sclerosis Patient-Derived Motor Neurons Cell Reports. ,vol. 7, pp. 1- 11 ,(2014) , 10.1016/J.CELREP.2014.03.019