Induced pluripotent stem cells from a spinal muscular atrophy patient AD Ebert, J Yu, FF Rose Jr, VB Mattis, CL Lorson, JA Thomson, ... Nature 457 (7227), 277-280, 2009 | 1933 | 2009 |
Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes Hd iPsc Consortium Cell stem cell 11 (2), 264-278, 2012 | 489 | 2012 |
Developmental alterations in Huntington's disease neural cells and pharmacological rescue in cells and mice Nature neuroscience 20 (5), 648-660, 2017 | 185 | 2017 |
A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels EC Wolstencroft, V Mattis, AA Bajer, PJ Young, CL Lorson Human Molecular Genetics 14 (9), 1199-1210, 2005 | 163 | 2005 |
Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy FF Rose Jr, VB Mattis, H Rindt, CL Lorson Human molecular genetics 18 (6), 997-1005, 2009 | 137 | 2009 |
Induced pluripotent stem cells: a new revolution for clinical neurology? VB Mattis, CN Svendsen The Lancet Neurology 10 (4), 383-394, 2011 | 132 | 2011 |
Targeting ATM ameliorates mutant Huntingtin toxicity in cell and animal models of Huntington’s disease XH Lu, VB Mattis, N Wang, I Al-Ramahi, N van den Berg, SA Fratantoni, ... Science translational medicine 6 (268), 268ra178-268ra178, 2014 | 128 | 2014 |
EZ spheres: a stable and expandable culture system for the generation of pre-rosette multipotent stem cells from human ESCs and iPSCs AD Ebert, BC Shelley, AM Hurley, M Onorati, V Castiglioni, TN Patitucci, ... Stem cell research 10 (3), 417-427, 2013 | 123 | 2013 |
HD iPSC-derived neural progenitors accumulate in culture and are susceptible to BDNF withdrawal due to glutamate toxicity VB Mattis, C Tom, S Akimov, J Saeedian, ME Østergaard, AL Southwell, ... Human molecular genetics 24 (11), 3257-3271, 2015 | 121 | 2015 |
Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts VB Mattis, R Rai, J Wang, CWT Chang, T Coady, CL Lorson Human genetics 120, 589-601, 2006 | 120 | 2006 |
Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model VB Mattis, AD Ebert, MY Fosso, CW Chang, CL Lorson Human molecular genetics 18 (20), 3906-3913, 2009 | 113 | 2009 |
Human Huntington’s disease iPSC-derived cortical neurons display altered transcriptomics, morphology, and maturation SR Mehta, CM Tom, Y Wang, C Bresee, D Rushton, PP Mathkar, J Tang, ... Cell reports 25 (4), 1081-1096. e6, 2018 | 96 | 2018 |
Huntington’s disease patient-derived astrocytes display electrophysiological impairments and reduced neuronal support VJ Garcia, DJ Rushton, CM Tom, ND Allen, PJ Kemp, CN Svendsen, ... Frontiers in neuroscience 13, 669, 2019 | 56 | 2019 |
A SMNΔ7 read-through product confers functionality to the SMNΔ7 protein VB Mattis, M Bowerman, R Kothary, CL Lorson Neuroscience letters 442 (1), 54-58, 2008 | 50 | 2008 |
Modeling Huntington׳ s disease with patient-derived neurons VB Mattis, CN Svendsen Brain Research 1656, 76-87, 2017 | 47 | 2017 |
Bioenergetic deficits in Huntington’s disease iPSC-derived neural cells and rescue with glycolytic metabolites Human molecular genetics 29 (11), 1757-1771, 2020 | 41 | 2020 |
Optimizing maturity and dose of iPSC-derived dopamine progenitor cell therapy for Parkinson’s disease BM Hiller, DJ Marmion, CA Thompson, NA Elliott, H Federoff, P Brundin, ... NPJ Regenerative medicine 7 (1), 24, 2022 | 38 | 2022 |
A patient-derived cellular model for Huntington’s disease reveals phenotypes at clinically relevant CAG lengths CLK Hung, T Maiuri, LE Bowie, R Gotesman, S Son, M Falcone, ... Molecular Biology of the Cell 29 (23), 2809-2820, 2018 | 37 | 2018 |
Neonatal immune-tolerance in mice does not prevent xenograft rejection VB Mattis, DR Wakeman, C Tom, HB Dodiya, SY Yeung, AH Tran, ... Experimental neurology 254, 90-98, 2014 | 37 | 2014 |
Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA VB Mattis, MY Fosso, CW Chang, CL Lorson BMC neuroscience 10, 1-6, 2009 | 35 | 2009 |