Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy C Long, L Amoasii, AA Mireault, JR McAnally, H Li, E Sanchez-Ortiz, ... Science 351 (6271), 400-403, 2016 | 1160 | 2016 |
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy L Amoasii, JCW Hildyard, H Li, E Sanchez-Ortiz, A Mireault, D Caballero, ... Science 362 (6410), 86-91, 2018 | 576 | 2018 |
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy L Amoasii, C Long, H Li, AA Mireault, JM Shelton, E Sanchez-Ortiz, ... Science translational medicine 9 (418), eaan8081, 2017 | 265 | 2017 |
CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells YL Min, H Li, C Rodriguez-Caycedo, AA Mireault, J Huang, JM Shelton, ... Science advances 5 (3), eaav4324, 2019 | 253 | 2019 |
Myotubularin controls desmin intermediate filament architecture and mitochondrial dynamics in human and mouse skeletal muscle K Hnia, H Tronchère, KK Tomczak, L Amoasii, P Schultz, AH Beggs, ... The Journal of clinical investigation 121 (1), 70-85, 2011 | 167 | 2011 |
MED12 regulates HSC-specific enhancers independently of mediator kinase activity to control hematopoiesis B Aranda-Orgilles, R Saldaña-Meyer, E Wang, E Trompouki, A Fassl, ... Cell stem cell 19 (6), 784-799, 2016 | 123 | 2016 |
Increased expression of wild-type or a centronuclear myopathy mutant of dynamin 2 in skeletal muscle of adult mice leads to structural defects and muscle weakness BS Cowling, A Toussaint, L Amoasii, P Koebel, A Ferry, L Davignon, ... The American journal of pathology 178 (5), 2224-2235, 2011 | 110 | 2011 |
Myotubularin and PtdIns3P remodel the sarcoplasmic reticulum in muscle in vivo L Amoasii, K Hnia, G Chicanne, A Brech, BS Cowling, MM Müller, ... Journal of cell science 126 (8), 1806-1819, 2013 | 80 | 2013 |
Receptor activator of NF-κB (RANK) stimulates the proliferation of epithelial cells of the epidermo-pilosebaceous unit V Duheron, E Hess, M Duval, M Decossas, B Castaneda, JE Klöpper, ... Proceedings of the National Academy of Sciences 108 (13), 5342-5347, 2011 | 75 | 2011 |
Lack of myotubularin (MTM1) leads to muscle hypotrophy through unbalanced regulation of the autophagy and ubiquitin‐proteasome pathways L Al‐Qusairi, I Prokic, L Amoasii, C Kretz, N Messaddeq, JL Mandel, ... The FASEB Journal 27 (8), 3384-3394, 2013 | 74 | 2013 |
Phosphatase-dead myotubularin ameliorates X-linked centronuclear myopathy phenotypes in mice L Amoasii, DL Bertazzi, H Tronchère, K Hnia, G Chicanne, B Rinaldi, ... Public Library of Science 8 (10), e1002965, 2012 | 65 | 2012 |
Myotubularin phosphoinositide phosphatases in human diseases L Amoasii, K Hnia, J Laporte Phosphoinositides and Disease, 209-233, 2012 | 55 | 2012 |
Genome editing of monogenic neuromuscular diseases: a systematic review C Long, L Amoasii, R Bassel-Duby, EN Olson JAMA neurology 73 (11), 1349-1355, 2016 | 48 | 2016 |
A MED13-dependent skeletal muscle gene program controls systemic glucose homeostasis and hepatic metabolism L Amoasii, W Holland, E Sanchez-Ortiz, KK Baskin, M Pearson, ... Genes & development 30 (4), 434-446, 2016 | 42 | 2016 |
NURR1 activation in skeletal muscle controls systemic energy homeostasis L Amoasii, E Sanchez-Ortiz, T Fujikawa, JK Elmquist, R Bassel-Duby, ... Proceedings of the National Academy of Sciences 116 (23), 11299-11308, 2019 | 36 | 2019 |
Loss of catalytically inactive lipid phosphatase myotubularin-related protein 12 impairs myotubularin stability and promotes centronuclear myopathy in zebrafish VA Gupta, K Hnia, LL Smith, SR Gundry, JE McIntire, J Shimazu, JR Bass, ... PLoS genetics 9 (6), e1003583, 2013 | 35 | 2013 |
In vivo non-invasive monitoring of dystrophin correction in a new Duchenne muscular dystrophy reporter mouse L Amoasii, H Li, Y Zhang, YL Min, E Sanchez-Ortiz, JM Shelton, C Long, ... Nature communications 10 (1), 4537, 2019 | 33 | 2019 |
Primary T-tubule and autophagy defects in the phosphoinositide phosphatase Jumpy/MTMR14 knockout mice muscle K Hnia, C Kretz, L Amoasii, J Böhm, X Liu, N Messaddeq, CK Qu, ... Advances in biological regulation 52 (1), 98-107, 2012 | 32 | 2012 |
Dmd reporter models containing humanized duchenne muscular dystrophy mutations L Amoasii, L Chengzu, R Bassel-Duby, E Olson US Patent App. 16/467,445, 2019 | 19 | 2019 |
Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences L Amoasii, E Olson | 19 | 2019 |