| Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice N Wein, A Vulin, MS Falzarano, CAK Szigyarto, B Maiti, A Findlay, ... Nature medicine 20 (9), 992-1000, 2014 | 131 | 2014 |
| Genetics and emerging treatments for Duchenne and Becker muscular dystrophy N Wein, L Alfano, KM Flanigan Pediatric Clinics 62 (3), 723-742, 2015 | 107 | 2015 |
| Efficient bypass of mutations in dysferlin deficient patient cells by antisense‐induced exon skipping N Wein, A Avril, M Bartoli, C Beley, S Chaouch, P Laforêt, A Behin, ... Human mutation 31 (2), 136-142, 2010 | 107 | 2010 |
| A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy M Krahn, N Wein, M Bartoli, W Lostal, S Courrier, N Bourg-Alibert, ... Science translational medicine 2 (50), 50ra69-50ra69, 2010 | 104 | 2010 |
| Personalized gene and cell therapy for Duchenne muscular dystrophy F Barthélémy, N Wein Neuromuscular Disorders 28 (10), 803-824, 2018 | 58 | 2018 |
| UMD‐DYSF, a novel locus specific database for the compilation and interactive analysis of mutations in the dysferlin gene G Blandin, C Beroud, V Labelle, K Nguyen, N Wein, D Hamroun, ... Human mutation 33 (3), E2317-E2331, 2012 | 54 | 2012 |
| Translational research and therapeutic perspectives in dysferlinopathies F Barthélémy, N Wein, M Krahn, N Lévy, M Bartoli Molecular Medicine 17, 875-882, 2011 | 50 | 2011 |
| Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45 AR Findlay, N Wein, Y Kaminoh, LE Taylor, DM Dunn, JR Mendell, ... Annals of neurology 77 (4), 668-674, 2015 | 43 | 2015 |
| The ZZ domain of dystrophin in DMD: making sense of missense mutations A Vulin, N Wein, DM Strandjord, EK Johnson, AR Findlay, B Maiti, ... Human mutation 35 (2), 257-264, 2014 | 38 | 2014 |
| Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells E Massouridès, J Polentes, PE Mangeot, V Mournetas, J Nectoux, ... Skeletal Muscle 5, 1-18, 2015 | 36 | 2015 |
| Exon 32 skipping of dysferlin rescues membrane repair in patients’ cells F Barthélémy, C Blouin, N Wein, V Mouly, S Courrier, E Dionnet, ... Journal of Neuromuscular Diseases 2 (3), 281-290, 2015 | 36 | 2015 |
| Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping LV Gushchina, EC Frair, N Rohan, AJ Bradley, TR Simmons, HD Chavan, ... Human Gene Therapy 32 (17-18), 882-894, 2021 | 33 | 2021 |
| The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development A Vulin, N Wein, TR Simmons, AM Rutherford, AR Findlay, JA Yurkoski, ... Neuromuscular Disorders 25 (11), 827-834, 2015 | 33 | 2015 |
| Efficient skipping of single exon duplications in DMD patient-derived cell lines using an antisense oligonucleotide approach N Wein, A Vulin, AR Findlay, F Gumienny, N Huang, SD Wilton, ... Journal of neuromuscular diseases 4 (3), 199-207, 2017 | 32 | 2017 |
| Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping TR Simmons, TA Vetter, N Huang, A Vulin-Chaffiol, N Wein, KM Flanigan Molecular Therapy-Methods & Clinical Development 21, 325-340, 2021 | 24 | 2021 |
| Designed U7 snRNAs inhibit DUX4 expression and improve FSHD-associated outcomes in DUX4 overexpressing cells and FSHD patient myotubes A Rashnonejad, G Amini-Chermahini, NK Taylor, N Wein, SQ Harper Molecular Therapy-Nucleic Acids 23, 476-486, 2021 | 24 | 2021 |
| Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy MA Waldrop, SA Moore, KD Mathews, BW Darbro, L Medne, R Finkel, ... Human mutation 43 (4), 511-528, 2022 | 23 | 2022 |
| Identification of different genomic deletions and one duplication in the dysferlin gene using multiplex ligation-dependent probe amplification and genomic quantitative PCR M Krahn, A Borges, C Navarro, R Schuit, T Stojkovic, Y Torrente, N Wein, ... Genetic testing and molecular biomarkers 13 (4), 439-442, 2009 | 23 | 2009 |
| Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse N Wein, TA Vetter, A Vulin, TR Simmons, EC Frair, AJ Bradley, ... Molecular Therapy-Methods & Clinical Development 26, 279-293, 2022 | 21 | 2022 |
| Immunolabelling and flow cytometry as new tools to explore dysferlinopathies N Wein, M Krahn, S Courrier, M Bartoli, E Salort-Campana, K Nguyen, ... Neuromuscular Disorders 20 (1), 57-60, 2010 | 20 | 2010 |
