| Single-dose gene-replacement therapy for spinal muscular atrophy JR Mendell, S Al-Zaidy, R Shell, WD Arnold, LR Rodino-Klapac, TW Prior, ... New England Journal of Medicine 377 (18), 1713-1722, 2017 | 2387 | 2017 |
| Eteplirsen for the treatment of Duchenne muscular dystrophy JR Mendell, LR Rodino‐Klapac, Z Sahenk, K Roush, L Bird, LP Lowes, ... Annals of neurology 74 (5), 637-647, 2013 | 898 | 2013 |
| Dystrophin immunity in Duchenne's muscular dystrophy JR Mendell, K Campbell, L Rodino-Klapac, Z Sahenk, C Shilling, S Lewis, ... New England Journal of Medicine 363 (15), 1429-1437, 2010 | 775 | 2010 |
| Current clinical applications of in vivo gene therapy with AAVs JR Mendell, SA Al-Zaidy, LR Rodino-Klapac, K Goodspeed, SJ Gray, ... Molecular Therapy 29 (2), 464-488, 2021 | 624 | 2021 |
| Longitudinal effect of eteplirsen versus historical control on ambulation in D uchenne muscular dystrophy JR Mendell, N Goemans, LP Lowes, LN Alfano, K Berry, J Shao, EM Kaye, ... Annals of neurology 79 (2), 257-271, 2016 | 542 | 2016 |
| Gentamicin‐induced readthrough of stop codons in Duchenne muscular dystrophy V Malik, LR Rodino‐Klapac, L Viollet, C Wall, W King, R Al‐Dahhak, ... Annals of neurology 67 (6), 771-780, 2010 | 369 | 2010 |
| Assessment of systemic delivery of rAAVrh74. MHCK7. micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial JR Mendell, Z Sahenk, K Lehman, C Nease, LP Lowes, NF Miller, ... JAMA neurology 77 (9), 1122-1131, 2020 | 346 | 2020 |
| A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy JR Mendell, Z Sahenk, V Malik, AM Gomez, KM Flanigan, LP Lowes, ... Molecular Therapy 23 (1), 192-201, 2015 | 290 | 2015 |
| Sustained alpha‐sarcoglycan gene expression after gene transfer in limb‐girdle muscular dystrophy, type 2D JR Mendell, LR Rodino‐Klapac, XQ Rosales, BD Coley, G Galloway, ... Annals of neurology 68 (5), 629-638, 2010 | 287 | 2010 |
| Limb‐girdle muscular dystrophy type 2D gene therapy restores α‐sarcoglycan and associated proteins JR Mendell, LR Rodino‐Klapac, X Rosales‐Quintero, J Kota, BD Coley, ... Annals of Neurology: Official Journal of the American Neurological …, 2009 | 268 | 2009 |
| Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease LR Rodino‐Klapac, AM Haidet, J Kota, C Handy, BK Kaspar, JR Mendell Muscle & Nerve: Official Journal of the American Association of …, 2009 | 245 | 2009 |
| Eteplirsen treatment for Duchenne muscular dystrophy: exon skipping and dystrophin production JS Charleston, FJ Schnell, J Dworzak, C Donoghue, S Lewis, L Chen, ... Neurology 90 (24), e2146-e2154, 2018 | 235 | 2018 |
| Follistatin gene delivery enhances muscle growth and strength in nonhuman primates J Kota, CR Handy, AM Haidet, CL Montgomery, A Eagle, ... Science translational medicine 1 (6), 6ra15-6ra15, 2009 | 223 | 2009 |
| Gene therapy for muscular dystrophy: lessons learned and path forward JR Mendell, L Rodino-Klapac, Z Sahenk, V Malik, BK Kaspar, CM Walker, ... Neuroscience letters 527 (2), 90-99, 2012 | 192 | 2012 |
| Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery LG Chicoine, CL Montgomery, WG Bremer, KM Shontz, DA Griffin, ... Molecular Therapy 22 (2), 338-347, 2014 | 178 | 2014 |
| A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy LR Rodino-Klapac, PML Janssen, CL Montgomery, BD Coley, ... Journal of translational medicine 5, 1-11, 2007 | 140 | 2007 |
| Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery LR Rodino-Klapac, CL Montgomery, WG Bremer, KM Shontz, V Malik, ... Molecular Therapy 18 (1), 109-117, 2010 | 139 | 2010 |
| Follistatin gene therapy for sporadic inclusion body myositis improves functional outcomes JR Mendell, Z Sahenk, S Al-Zaidy, LR Rodino-Klapac, LP Lowes, ... Molecular Therapy 25 (4), 870-879, 2017 | 136 | 2017 |
| 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 | 130 | 2014 |
| Emerging drugs for Duchenne muscular dystrophy V Malik, LR Rodino-Klapac, JR Mendell Expert opinion on emerging drugs 17 (2), 261-277, 2012 | 124 | 2012 |