DUX4, a candidate gene for facioscapulohumeral muscular dystrophy, causes p53‐dependent myopathy in vivo LM Wallace, SE Garwick, W Mei, A Belayew, F Coppee, KJ Ladner, ... Annals of neurology 69 (3), 540-552, 2011 | 271 | 2011 |
RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy LM Wallace, J Liu, JS Domire, SE Garwick-Coppens, SM Guckes, ... Molecular Therapy 20 (7), 1417-1423, 2012 | 118 | 2012 |
AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD CR Giesige, LM Wallace, KN Heller, JO Eidahl, NY Saad, AM Fowler, ... JCI insight 3 (22), 2018 | 64 | 2018 |
High mobility group box-1 (HMGB1) is increased in injured mouse spinal cord and can elicit neurotoxic inflammation KA Kigerl, W Lai, LM Wallace, H Yang, PG Popovich Brain, behavior, and immunity 72, 22-33, 2018 | 61 | 2018 |
Pre-clinical safety and off-target studies to support translation of AAV-mediated RNAi therapy for FSHD LM Wallace, NY Saad, NK Pyne, AM Fowler, JO Eidahl, JS Domire, ... Molecular Therapy-Methods & Clinical Development 8, 121-130, 2018 | 57 | 2018 |
Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models KH Morelli, LB Griffin, NK Pyne, LM Wallace, AM Fowler, SN Oprescu, ... The Journal of clinical investigation 129 (12), 2019 | 55 | 2019 |
Recombinant virus products and methods for inhibition of expression of DUX4 SQ Harper, J Liu, SG Coppens, L Wallace US Patent 9,469,851, 2016 | 52 | 2016 |
RNA interference improves myopathic phenotypes in mice over-expressing FSHD region gene 1 (FRG1) LM Wallace, SE Garwick-Coppens, R Tupler, SQ Harper Molecular Therapy 19 (11), 2048-2054, 2011 | 52 | 2011 |
PP2A: B56ϵ, a substrate of caspase-3, regulates p53-dependent and p53-independent apoptosis during development Z Jin, L Wallace, SQ Harper, J Yang Journal of Biological Chemistry 285 (45), 34493-34502, 2010 | 52 | 2010 |
Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4 JO Eidahl, CR Giesige, JS Domire, LM Wallace, AM Fowler, SM Guckes, ... Human molecular genetics 25 (20), 4577-4589, 2016 | 51 | 2016 |
Recombinant virus products and methods for inhibition of expression of DUX4 SQ HARPER, J Liu, S Coppens, L Wallace US Patent 10,301,649, 2019 | 46* | 2019 |
Considerations for the use of Cre recombinase for conditional gene deletion in the mouse lens PT Lam, SL Padula, TV Hoang, JE Poth, L Liu, C Liang, AS LeFever, ... Human genomics 13 (1), 10, 2019 | 30 | 2019 |
Recombinant virus products and methods for inhibition of expression of DUX4 SQ Harper, J Liu, S Coppens, L Wallace US Patent 10,301,649, 2019 | 26 | 2019 |
Dose-dependent toxicity of humanized Renilla reniformis GFP (hrGFP) limits its utility as a reporter gene in mouse muscle LM Wallace, A Moreo, KR Clark, SQ Harper Molecular Therapy-Nucleic Acids 2, e86, 2013 | 20 | 2013 |
Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences E Ansseau, JS Domire, LM Wallace, JO Eidahl, SM Guckes, CR Giesige, ... PLoS One 10 (3), e0118813, 2015 | 17 | 2015 |
RNAi-mediated gene silencing of mutant myotilin improves myopathy in LGMD1A mice J Liu, LM Wallace, SE Garwick-Coppens, DD Sloboda, CS Davis, ... Molecular Therapy-Nucleic Acids 3, e160, 2014 | 17 | 2014 |
RNAi therapy for dominant muscular dystrophies and other myopathies LM Wallace, SE Garwick, SQ Harper Muscle Gene Therapy, 99-115, 2010 | 12 | 2010 |
Rapid cloning and validation of microRNA shuttle vectors: a practical guide RL Boudreau, SE Garwick-Coppens, J Liu, LM Wallace, SQ Harper RNA Interference Techniques, 19-37, 2011 | 10 | 2011 |
O. 8DUX4 mRNA silencing with CRISPR-Cas13 gene therapy as a prospective treatment for Facioscapulohumeral muscular dystrophy A Rashnonejad, GA Chermahini, L Wallace, S Harper Neuromuscular Disorders 29, S40, 2019 | 7 | 2019 |
Muscle Gene Therapy LM Wallace, SE Garwick, SQ Harper, D Duan | 6 | 2010 |