| Year |
Citation |
Score |
| 2024 |
Mendell JR, Pozsgai ER, Lewis S, Griffin DA, Lowes LP, Alfano LN, Lehman KJ, Church K, Reash NF, Iammarino MA, Sabo B, Potter R, Neuhaus S, Li X, Stevenson H, ... Rodino-Klapac LR, et al. Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results. Nature Medicine. PMID 38177855 DOI: 10.1038/s41591-023-02730-9 |
0.376 |
|
| 2023 |
Potter RA, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR. Expression and function of Four AAV-based constructs for dystrophin restoration in the mdx mouse model of duchenne muscular dystrophy. Biology Open. PMID 37670674 DOI: 10.1242/bio.059797 |
0.314 |
|
| 2023 |
Seo YE, Baine SH, Kempton AN, Rogers OC, Lewis S, Adegboye K, Haile A, Griffin DA, Peterson EL, Pozsgai ER, Potter RA, Rodino-Klapac LR. Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model. Molecular Therapy. Methods & Clinical Development. 28: 284-299. PMID 36816759 DOI: 10.1016/j.omtm.2023.01.004 |
0.403 |
|
| 2021 |
Crowe KE, Zygmunt DA, Heller K, Rodino-Klapac L, Noguchi S, Nishino I, Martin PT. Visualizing Muscle Sialic Acid Expression in the GNED207VTgGne-/- Cmah-/- Model of GNE Myopathy: A Comparison of Dietary and Gene Therapy Approaches. Journal of Neuromuscular Diseases. PMID 34511508 DOI: 10.3233/JND-200575 |
0.334 |
|
| 2021 |
Potter RA, Griffin DA, Heller KN, Peterson EL, Clark EK, Mendell JR, Rodino-Klapac LR. Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the Mouse Model of Duchenne Muscular Dystrophy. Human Gene Therapy. PMID 33397205 DOI: 10.1089/hum.2019.255 |
0.327 |
|
| 2020 |
Griffin DA, Pozsgai ER, Heller KN, Potter RA, Peterson EL, Rodino-Klapac LR. Pre-clinical Systemic Delivery of Adeno-Associated Alpha-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy. Human Gene Therapy. PMID 33349138 DOI: 10.1089/hum.2019.199 |
0.406 |
|
| 2020 |
Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, Iammarino MA, Alfano LN, Nicholl A, Al-Zaidy S, Lewis S, Church K, Shell R, Cripe LH, Potter RA, ... ... Rodino-Klapac LR, et al. Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial. Jama Neurology. PMID 32539076 DOI: 10.1001/Jamaneurol.2020.1484 |
0.429 |
|
| 2020 |
Asher DR, Thapa K, Dharia SD, Khan N, Potter RA, Rodino-Klapac LR, Mendell JR. Clinical Development on the Frontier: Gene Therapy for Duchenne Muscular Dystrophy. Expert Opinion On Biological Therapy. PMID 32031420 DOI: 10.1080/14712598.2020.1725469 |
0.413 |
|
| 2019 |
Alfano LN, Charleston JS, Connolly AM, Cripe L, Donoghue C, Dracker R, Dworzak J, Eliopoulos H, Frank DE, Lewis S, Lucas K, Lynch J, Milici AJ, Flynt A, Naughton E, ... Rodino-Klapac LR, et al. Long-term treatment with eteplirsen in nonambulatory patients with Duchenne muscular dystrophy. Medicine. 98: e15858. PMID 31261494 DOI: 10.1097/Md.0000000000015858 |
0.327 |
|
| 2019 |
Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson EL, Griffin DA, Church K, Cheatham S, ... ... Rodino-Klapac LR, et al. Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D (LGMD2D). Human Gene Therapy. PMID 30838895 DOI: 10.1089/Hum.2019.006 |
0.477 |
|
| 2019 |
Potter R, Griffin D, Heller K, Mendell J, Rodino-Klapac L. P.312Functional and histological improvements comparing 4 micro-dystrophin constructs in the mdx mouse model of DMD Neuromuscular Disorders. 29: S158. DOI: 10.1016/J.Nmd.2019.06.426 |
0.306 |
|
| 2019 |
Iammarino M, Miller N, Alfano L, Lehman K, Rodino-Klapac L, Mendell J, Lowes L. P.174Establishing divergent phenotypes in limb girdle muscular dystrophies Neuromuscular Disorders. 29. DOI: 10.1016/J.Nmd.2019.06.229 |
0.322 |
|
| 2018 |
Giesige CR, Wallace LM, Heller KN, Eidahl JO, Saad NY, Fowler AM, Pyne NK, Al-Kharsan M, Rashnonejad A, Chermahini GA, Domire JS, Mukweyi D, Garwick-Coppens SE, Guckes SM, McLaughlin KJ, ... ... Rodino-Klapac LR, et al. AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD. Jci Insight. 3. PMID 30429376 DOI: 10.1172/Jci.Insight.123538 |
0.489 |
|
| 2018 |
Xu R, Jia Y, Zygmunt DA, Cramer ML, Crowe KE, Shao G, Maki AE, Guggenheim HN, Hood BC, Griffin DA, Peterson E, Bolon B, Cheatham JP, Cheatham SL, Flanigan KM, ... Rodino-Klapac LR, et al. An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK. to Leg Skeletal Muscles in the Rhesus Macaque. Molecular Therapy. Methods & Clinical Development. 10: 89-104. PMID 30073180 DOI: 10.1016/J.Omtm.2018.06.002 |
0.438 |
|
| 2018 |
Charleston JS, Schnell FJ, Dworzak J, Donoghue C, Lewis S, Chen L, Young GD, Milici AJ, Voss J, DeAlwis U, Wentworth B, Rodino-Klapac LR, Sahenk Z, Frank D, Mendell JR. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology. PMID 29752304 DOI: 10.1212/Wnl.0000000000005680 |
0.334 |
|
| 2018 |
Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ. Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD. Molecular Therapy. Methods & Clinical Development. 8: 121-130. PMID 29387734 DOI: 10.1016/J.Omtm.2017.12.005 |
0.395 |
|
| 2017 |
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, et al. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. The New England Journal of Medicine. 377: 1713-1722. PMID 29091557 DOI: 10.1056/Nejmoa1706198 |
0.339 |
|
| 2017 |
Potter RA, Griffin DA, Sondergaard PC, Johnson RW, Pozsgai ER, Heller KN, Peterson EL, Lehtimaki KK, Windish HP, Mittal PJ, Albrecht DE, Mendell JR, Rodino-Klapac LR. Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Functional Improvement for Dysferlinopathy. Human Gene Therapy. PMID 28707952 DOI: 10.1089/Hum.2017.062 |
0.507 |
|
| 2017 |
Heller KN, Mendell JT, Mendell JR, Rodino-Klapac LR. MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin. Jci Insight. 2. PMID 28469083 DOI: 10.1172/Jci.Insight.93309 |
0.417 |
|
| 2017 |
Cramer ML, Shao G, Rodino-Klapac LR, Chicoine LG, Martin PT. Induction of T-cell infiltration and programmed death ligand 2 expression by adeno-associated virus in rhesus macaque skeletal muscle and modulation by prednisone. Human Gene Therapy. PMID 28345428 DOI: 10.1089/Hum.2016.113 |
0.402 |
|
| 2017 |
Pozsgai ER, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR. Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28284983 DOI: 10.1016/J.Ymthe.2017.02.013 |
0.507 |
|
| 2017 |
Mendell JR, Sahenk Z, Al-Zaidy S, Rodino-Klapac LR, Lowes LP, Alfano LN, Berry K, Miller N, Yalvac M, Dvorchik I, Moore-Clingenpeel M, Flanigan KM, Church K, Shontz K, Curry C, et al. Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28279643 DOI: 10.1016/J.Ymthe.2017.02.015 |
0.411 |
|
| 2017 |
Mendell JR, Sahenk Z, Rodino-Klapac LR. Clinical trials of exon skipping in Duchenne muscular dystrophy Expert Opinion On Orphan Drugs. 5: 683-690. DOI: 10.1080/21678707.2017.1366310 |
0.41 |
|
| 2017 |
Schnell F, Donoghue C, Dworzak J, Charleston J, Frank D, Wilton S, Fletcher S, Lewis S, Mendell J, Rodino-Klapac L, Sahenk Z. Development of a validated Western blot method for quantification of human dystrophin protein used in phase 2 and 3 clinical trials of eteplirsen for the treatment of Duchenne muscular dystrophy Neuromuscular Disorders. 27: S16. DOI: 10.1016/S0960-8966(17)30264-X |
0.332 |
|
| 2017 |
Charleston J, Schnell F, Dworzak J, Donoghue C, Lewis S, Rodino-Klapac L, Sahenk Z, Voss J, DeAlwis U, Frank D, Mendell J. Long-term treatment with the phosphorodiamidate morpholino oligomer eteplirsen in patients with Duchenne muscular dystrophy amenable to exon 51 skipping: analysis of dystrophin production Neuromuscular Disorders. 27: S16. DOI: 10.1016/S0960-8966(17)30263-8 |
0.307 |
|
| 2017 |
Lowes L, Al-Zaidy S, Shell R, Arnold W, Rodino-Klapac L, Prior T, Alfano L, Berry K, Church K, Kissel J, Nagendran S, L'Italien J, Sproule D, Wells C, Burghes A, et al. P.374 - AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: patients treated early with the proposed therapeutic dose were able to sit unassisted at a younger age Neuromuscular Disorders. 27. DOI: 10.1016/J.Nmd.2017.06.414 |
0.368 |
|
| 2017 |
Shell R, Al-Zaidy S, Arnold W, Rodino-Klapac L, Prior T, Lowes L, Alfano L, Berry K, Church K, Kissel J, Nagendran S, L'Italien J, Sproule D, Wells C, Burghes A, et al. AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: decreased need of ventilatory and nutritional support at End-of-Study Neuromuscular Disorders. 27: S208. DOI: 10.1016/J.Nmd.2017.06.413 |
0.361 |
|
| 2017 |
Mendell J, Al-Zaidy S, Shell R, Arnold W, Rodino-Klapac L, Prior T, Lowes L, Alfano L, Berry K, Church K, Kissel J, Nagendran S, L'Italien J, Sproule D, Wells C, et al. P.372 - AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: end-of-Study event free survival and achievement of developmental milestones Neuromuscular Disorders. 27. DOI: 10.1016/J.Nmd.2017.06.412 |
0.351 |
|
| 2017 |
Wallace L, Griffin D, Pyne N, Domire J, Rodino-Klapac L, Harper S. P.340 - Translating DUX4-targeted RNAi therapy for Facioscapulohumeral muscular dystrophy Neuromuscular Disorders. 27. DOI: 10.1016/J.Nmd.2017.06.380 |
0.311 |
|
| 2017 |
Wein N, Simmons T, Gumienny F, Huang N, Heller K, Yurkoski J, Rodino-Klapac L, Muntoni F, Flanigan K. A single neonatal injection of an AAV9.U7snRNA virus mediating skipping of dmd exon 2 allows dystrophin expression preventing apparition of pathologic features in the Dup2 mouse one year post injection Neuromuscular Disorders. 27. DOI: 10.1016/J.Nmd.2017.06.339 |
0.305 |
|
| 2017 |
Shell R, Al-Zaidy S, Arnold WD, Rodino-Klapac L, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Burghes AH, Foust K, et al. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use European Journal of Paediatric Neurology. 21: e14. DOI: 10.1016/J.Ejpn.2017.04.1217 |
0.339 |
|
| 2017 |
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac L, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Cardenas J, et al. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones European Journal of Paediatric Neurology. 21: e13-e14. DOI: 10.1016/J.Ejpn.2017.04.1216 |
0.346 |
|
| 2017 |
Dworzak J, Frank DE, Charleston JS, Schnell FJ, Voss J, Donoghue C, Milici AJ, Aeffner F, Young GD, Moore SA, Lewis S, Peterson E, Sahenk Z, Shontz K, Rodino-Klapac LR, et al. Quantification of dystrophin to assess the effects of dystrophin-restoring treatments in Duchenne muscular dystrophy: Lessons from the development of eteplirsen European Journal of Paediatric Neurology. 21: e93-e94. DOI: 10.1016/J.Ejpn.2017.04.1209 |
0.34 |
|
| 2016 |
Mendell JR, Rodino-Klapac LR. CRISPR/Cas9 treatment for Duchenne muscular dystrophy. Cell Research. PMID 26926391 DOI: 10.1038/Cr.2016.28 |
0.395 |
|
| 2016 |
Griffin DA, Johnson RW, Whitlock JM, Pozsgai ER, Heller KN, Grose WE, Arnold WD, Sahenk Z, Hartzell HC, Rodino-Klapac LR. Defective membrane fusion and repair in Anoctamin5 -deficient muscular dystrophy. Human Molecular Genetics. PMID 26911675 DOI: 10.1093/Hmg/Ddw063 |
0.379 |
|
| 2016 |
Pozsgai E, Griffin D, Heller K, Mendell J, Rodino-Klapac L. 622. Systemic β-Sarcoglycan Gene Therapy for Treatment of Cardiac and Skeletal Muscle Deficits in LGMD2E Molecular Therapy. 24: S246-S247. DOI: 10.1016/S1525-0016(16)33430-X |
0.489 |
|
| 2016 |
Mendell JR, Sahenk Z, Hogan M, Al-Zaidy S, Flanigan K, Rodino-Klapac LR, McColly M, Church K, Lewis S, Lowes L, Alfano L, Berry K, Miller N, Dvorchik I, Moore-Clingenpeel M, et al. 497. Follistatin Gene Therapy Improves Six Minute Walk Distance in Sporadic Inclusion Body Myositis (sIBM) Molecular Therapy. 24: S197-S198. DOI: 10.1016/S1525-0016(16)33306-8 |
0.358 |
|
| 2016 |
Wallace LM, Domire JS, Griffin DA, Rodino-Klapac LR, Harper SQ. 386. Toxicology for DUX4-Targeted MicroRNAs Molecular Therapy. 24: S153. DOI: 10.1016/S1525-0016(16)33195-1 |
0.489 |
|
| 2016 |
Heller K, Meadows E, Lewis S, Mendell J, Mendell J, Rodino-Klapac L. 379. MicroRNA-29 and Micro-Dystrophin Combinatorial Therapy Suppresses Fibrosis and Restores Function to mdx/utrn+/− Mice Molecular Therapy. 24: S151. DOI: 10.1016/S1525-0016(16)33188-4 |
0.4 |
|
| 2016 |
Charleston J, Schnell F, Dworzak J, Donoghue C, Lewis S, Rodino-Klapac L, Sahenk Z, Shanks C, Voss J, DeAlwis U, Frank D, Wentworth B, Mendell J. Long-term treatment with eteplirsen promotes exon 51 skipping and novel dystrophin protein production in Duchenne muscular dystrophy patients Neuromuscular Disorders. 26: S153. DOI: 10.1016/J.Nmd.2016.06.246 |
0.316 |
|
| 2015 |
Al-Zaidy SA, Sahenk Z, Rodino-Klapac LR, Kaspar B, Mendell JR. Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy. Journal of Neuromuscular Diseases. 2: 185-192. PMID 27858738 DOI: 10.3233/Jnd-150083 |
0.47 |
|
| 2015 |
Pozsgai ER, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR. β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice. Gene Therapy. PMID 26214262 DOI: 10.1038/gt.2015.80 |
0.403 |
|
| 2015 |
Heller KN, Montgomery CL, Shontz KM, Clark KR, Mendell JR, Rodino-Klapac LR. Human α7 integrin gene (ITGΑ7) delivered by adeno-associated virus extends survival of severely affected dystrophin/utrophin deficient mice. Human Gene Therapy. PMID 26076707 DOI: 10.1089/hum.2015.062 |
0.397 |
|
| 2015 |
Sondergaard PC, Griffin DA, Pozsgai ER, Johnson RW, Grose WE, Heller KN, Shontz KM, Montgomery CL, Liu J, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models. Annals of Clinical and Translational Neurology. 2: 256-70. PMID 25815352 DOI: 10.1002/Acn3.172 |
0.525 |
|
| 2015 |
Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, Lowes LP, Alfano LN, Berry K, Meadows E, Lewis S, Braun L, Shontz K, Rouhana M, Clark KR, Rosales XQ, ... ... Rodino-Klapac LR, et al. A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 192-201. PMID 25322757 DOI: 10.1038/Mt.2014.200 |
0.449 |
|
| 2015 |
Pozsgai ER, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR. 506. β-Sarcoglycan Gene Transfer Prevents Muscle Fibrosis and Inflammation in an Aged LGMD2E Mouse Model Molecular Therapy. 23: S202-S203. DOI: 10.1016/S1525-0016(16)34115-6 |
0.498 |
|
| 2015 |
Simmons TR, Wein N, Vulin-Chaffiol A, Heller K, Rutherford A, Rodino-Klapac L, Flanigan KM. 505. Treatment of DMD 5’ Mutations Through Two Different exon2 Skipping Strategies: Intramuscular Delivery of rAAV9.snRNA Mediated Skipping and Antisense Morpholino Oligomers Molecular Therapy. 23: S202. DOI: 10.1016/S1525-0016(16)34114-4 |
0.464 |
|
| 2015 |
Mendell JR, Rodino-Klapac L, Sahenk Z, Rouch K, Bird L, Lowes L, Alfano L, Berry K, Lewis S, Shontz K, Flanigan K, Shilling C, Duda P, Saoud J. C-2. Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for the Treatment of Duchenne Muscular Dystrophy (DMD): 168 Week Update on Six-Minute Walk Test (6MWT), Pulmonary Function Testing (PFT), and Safety Molecular Therapy. 23: S16. DOI: 10.1016/S1525-0016(16)33641-3 |
0.414 |
|
| 2015 |
Kaye E, Mendell J, Rodino-Klapac L, Sahenk Z, Roush K, Lowes L, Alfano L, Gomez A, Malik V, Flanigan K, Shilling C, Duda P, Saoud J. PP09.16 – 2691: Eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD): 3.2 year update on six-minute walk test (6MWT), pulmonary function testing (PFT), and safety European Journal of Paediatric Neurology. 19: S69. DOI: 10.1016/S1090-3798(15)30228-2 |
0.373 |
|
| 2015 |
Kaye E, Mendell J, Rodino-Klapac L, Sahenk Z, Lowes L, Alfano L, Berry K, Gomez Ramirez A, Lewis S, Flanigan K, Cripe L, Al-Zaidy S, Duda P, Sazani P, Saoud J. Eteplirsen, a Phosphorodiamidate morpholino oligomer (PMO) for the treatment of Duchenne muscular dystrophy (DMD): Clinical update Neuromuscular Disorders. 25: S263. DOI: 10.1016/J.Nmd.2015.06.282 |
0.36 |
|
| 2014 |
Al-Zaidy S, Rodino-Klapac L, Mendell JR. Gene therapy for muscular dystrophy: Moving the field forward Pediatric Neurology. 51: 607-618. PMID 25439576 DOI: 10.1016/J.Pediatrneurol.2014.08.002 |
0.401 |
|
| 2014 |
Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Brioschi S, Bovolenta M, Neri M, ... ... Rodino-Klapac LR, et al. Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice. Nature Medicine. 20: 992-1000. PMID 25108525 DOI: 10.1038/Nm.3628 |
0.445 |
|
| 2014 |
Chicoine LG, Montgomery CL, Bremer WG, Shontz KM, Griffin DA, Heller KN, Lewis S, Malik V, Grose WE, Shilling CJ, Campbell KJ, Preston TJ, Coley BD, Martin PT, Walker CM, ... ... Rodino-Klapac LR, et al. Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 338-47. PMID 24196577 DOI: 10.1038/Mt.2013.244 |
0.34 |
|
| 2014 |
Sahenk Z, Galloway G, Clark KR, Malik V, Rodino-Klapac LR, Kaspar BK, Chen L, Braganza C, Montgomery C, Mendell JR. AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 511-21. PMID 24162799 DOI: 10.1038/Mt.2013.250 |
0.433 |
|
| 2014 |
Chicoine LG, Rodino-Klapac LR, Shao G, Xu R, Bremer WG, Camboni M, Golden B, Montgomery CL, Shontz K, Heller KN, Griffin DA, Lewis S, Coley BD, Walker CM, Clark KR, et al. Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 713-24. PMID 24145553 DOI: 10.1038/Mt.2013.246 |
0.596 |
|
| 2014 |
Heller K, Montgomery C, Shontz K, Clark K, Mendell J, Rodino-Klapac L. G.O.26 Neuromuscular Disorders. 24: 923. DOI: 10.1016/J.Nmd.2014.06.425 |
0.489 |
|
| 2014 |
Mendell J, Sahenk Z, Rodino-Klapac L, Clark K, Lewis K, Shontz K, Al-Zaidy S, Alfano L, Lowes, L, Berry K, Malik V, Shilling C, Rosales X, Walker C, et al. G.O.25 Neuromuscular Disorders. 24: 923. DOI: 10.1016/J.Nmd.2014.06.424 |
0.47 |
|
| 2014 |
Mendell J, Rodino-Klapac L, Sahenk Z, Roush K, Bird L, Lowes L, Alfano L, Gomez A, Lewis S, Malik V, Shontz K, Flanigan K, Shilling C, Sazani P, Saoud J, et al. G.O.24 Neuromuscular Disorders. 24: 922. DOI: 10.1016/J.Nmd.2014.06.423 |
0.395 |
|
| 2014 |
Griffin D, Pozsgai E, Johnson R, Grose W, Heller K, Mendell J, Sahenk Z, Rodino-Klapac L. G.P.289 Neuromuscular Disorders. 24: 904. DOI: 10.1016/J.Nmd.2014.06.365 |
0.482 |
|
| 2014 |
Pozsgai E, Griffin D, Heller K, Mendell J, Rodino-Klapac L. G.P.227 Neuromuscular Disorders. 24: 885. DOI: 10.1016/J.Nmd.2014.06.303 |
0.48 |
|
| 2014 |
Sondergaard P, Griffin D, Pozsgai E, Johnson R, Mendell J, Rodino-Klapac L. G.O.9 Neuromuscular Disorders. 24: 851-852. DOI: 10.1016/J.Nmd.2014.06.197 |
0.482 |
|
| 2014 |
Sazani P, Magee T, Charleston JS, Shanks C, Zhang J, Carver M, Rodino-Klapac L, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Malik V, et al. G.P.110 Neuromuscular Disorders. 24: 828. DOI: 10.1016/J.Nmd.2014.06.124 |
0.365 |
|
| 2014 |
Rodino-Klapac LR, Mendell JR. Principles and Practice of Molecular Therapies Neuromuscular Disorders of Infancy, Childhood, and Adolescence: a Clinician's Approach. 1035-1052. DOI: 10.1016/B978-0-12-417044-5.00051-2 |
0.329 |
|
| 2014 |
Sahenk Z, Rodino-Klapac LR. Dystrophinopathies Neuromuscular Disorders in Clinical Practice. 2147483647: 1207-1229. DOI: 10.1007/978-1-4614-6567-6_56 |
0.331 |
|
| 2013 |
Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Annals of Neurology. 74: 637-47. PMID 23907995 DOI: 10.1002/Ana.23982 |
0.337 |
|
| 2013 |
Rodino-Klapac LR, Janssen PM, Shontz KM, Canan B, Montgomery CL, Griffin D, Heller K, Schmelzer L, Handy C, Clark KR, Sahenk Z, Mendell JR, Kaspar BK. Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model. Human Molecular Genetics. 22: 4929-37. PMID 23863459 DOI: 10.1093/Hmg/Ddt342 |
0.48 |
|
| 2013 |
Rosales XQ, Malik V, Sneh A, Chen L, Lewis S, Kota J, Gastier-Foster JM, Astbury C, Pyatt R, Reshmi S, Rodino-Klapac LR, Clark KR, Mendell JR, Sahenk Z. Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation. Muscle & Nerve. 47: 731-9. PMID 23553538 DOI: 10.1002/Mus.23669 |
0.336 |
|
| 2013 |
Rodino-Klapac LR, Mendell JR, Sahenk Z. Update on the treatment of Duchenne muscular dystrophy. Current Neurology and Neuroscience Reports. 13: 332. PMID 23328943 DOI: 10.1007/S11910-012-0332-1 |
0.417 |
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| 2013 |
Heller KN, Montgomery CL, Janssen PM, Clark KR, Mendell JR, Rodino-Klapac LR. AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 520-5. PMID 23319059 DOI: 10.1038/Mt.2012.281 |
0.492 |
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| 2013 |
Mendell J, Rodino-Klapac L, Sahenk Z, Roush K, Bird L, Lowes L, Alfano L, Gomez A, Lewis S, Malik V, Shontz K, Flanigan K, Kean J, Allen H, Shilling C, et al. P.11.20 Results at 2years of a phase IIb extension study of the exon-skipping drug eteplirsen in patients with DMD Neuromuscular Disorders. 23: 804-805. DOI: 10.1016/J.Nmd.2013.06.581 |
0.394 |
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| 2012 |
Grose WE, Clark KR, Griffin D, Malik V, Shontz KM, Montgomery CL, Lewis S, Brown RH, Janssen PM, Mendell JR, Rodino-Klapac LR. Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer. Plos One. 7: e39233. PMID 22720081 DOI: 10.1371/Journal.Pone.0039233 |
0.491 |
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| 2012 |
Malik V, Rodino-Klapac LR, Mendell JR. Emerging drugs for Duchenne muscular dystrophy. Expert Opinion On Emerging Drugs. 17: 261-77. PMID 22632414 DOI: 10.1517/14728214.2012.691965 |
0.35 |
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| 2012 |
Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR. Gene therapy for muscular dystrophy: lessons learned and path forward. Neuroscience Letters. 527: 90-9. PMID 22609847 DOI: 10.1016/J.Neulet.2012.04.078 |
0.5 |
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| 2012 |
Grose W, Griffin D, Shontz K, Malik V, Montgomery C, Janssen P, Clark R, Mendell J, Rodino-Klapac L. Correction of Membrane Repair Defects in Dysferlin Deficient Muscle Following AAV5 Gene Transfer (S55.006) Neurology. 78: S55.006-S55.006. DOI: 10.1212/Wnl.78.1_Meetingabstracts.S55.006 |
0.396 |
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| 2012 |
Meadows E, Kota J, Lewis S, Sahenk Z, Clark R, Mendell J, Rodino-Klapac L. Reducing Skeletal Muscle Fibrosis with AAV-Delivered miR-29 (P04.089) Neurology. 78: P04.089-P04.089. DOI: 10.1212/Wnl.78.1_Meetingabstracts.P04.089 |
0.357 |
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| 2011 |
Beastrom N, Lu H, Macke A, Canan BD, Johnson EK, Penton CM, Kaspar BK, Rodino-Klapac LR, Zhou L, Janssen PM, Montanaro F. mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx Mice. The American Journal of Pathology. 179: 2464-74. PMID 21893021 DOI: 10.1016/J.Ajpath.2011.07.009 |
0.422 |
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| 2011 |
Rodino-Klapac LR, Montgomery CL, Mendell JR, Chicoine LG. AAV-mediated gene therapy to the isolated limb in rhesus macaques. Methods in Molecular Biology (Clifton, N.J.). 709: 287-98. PMID 21194036 DOI: 10.1007/978-1-61737-982-6_19 |
0.45 |
|
| 2010 |
Malik V, Rodino-Klapac LR, Viollet L, Mendell JR. Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy. Therapeutic Advances in Neurological Disorders. 3: 379-89. PMID 21179598 DOI: 10.1177/1756285610388693 |
0.381 |
|
| 2010 |
Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, et al. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Annals of Neurology. 68: 629-38. PMID 21031578 DOI: 10.1002/Ana.22251 |
0.392 |
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| 2010 |
Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, et al. Dystrophin immunity in Duchenne's muscular dystrophy. The New England Journal of Medicine. 363: 1429-37. PMID 20925545 DOI: 10.1056/Nejmoa1000228 |
0.421 |
|
| 2010 |
Malik V, Rodino-Klapac LR, Viollet L, Wall C, King W, Al-Dahhak R, Lewis S, Shilling CJ, Kota J, Serrano-Munuera C, Hayes J, Mahan JD, Campbell KJ, Banwell B, Dasouki M, et al. Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Annals of Neurology. 67: 771-80. PMID 20517938 DOI: 10.1002/Ana.22024 |
0.354 |
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| 2010 |
Mendell JR, Rodino-Klapac LR, Malik V. Molecular therapeutic strategies targeting Duchenne muscular dystrophy. Journal of Child Neurology. 25: 1145-8. PMID 20498331 DOI: 10.1177/0883073810371005 |
0.43 |
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| 2010 |
Rodino-Klapac LR, Montgomery CL, Bremer WG, Shontz KM, Malik V, Davis N, Sprinkle S, Campbell KJ, Sahenk Z, Clark KR, Walker CM, Mendell JR, Chicoine LG. Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 109-17. PMID 19904237 DOI: 10.1038/Mt.2009.254 |
0.439 |
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| 2009 |
Kota J, Handy CR, Haidet AM, Montgomery CL, Eagle A, Rodino-Klapac LR, Tucker D, Shilling CJ, Therlfall WR, Walker CM, Weisbrode SE, Janssen PM, Clark KR, Sahenk Z, Mendell JR, et al. Follistatin gene delivery enhances muscle growth and strength in nonhuman primates. Science Translational Medicine. 1: 6ra15. PMID 20368179 DOI: 10.1126/Scitranslmed.3000112 |
0.489 |
|
| 2009 |
Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, et al. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Annals of Neurology. 66: 290-7. PMID 19798725 DOI: 10.1002/Ana.21732 |
0.406 |
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| 2009 |
Rodino-Klapac LR, Haidet AM, Kota J, Handy C, Kaspar BK, Mendell JR. Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease. Muscle & Nerve. 39: 283-96. PMID 19208403 DOI: 10.1002/Mus.21244 |
0.484 |
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| 2009 |
Martin PT, Xu R, Rodino-Klapac LR, Oglesbay E, Camboni M, Montgomery CL, Shontz K, Chicoine LG, Clark KR, Sahenk Z, Mendell JR, Janssen PM. Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice. American Journal of Physiology. Cell Physiology. 296: C476-88. PMID 19109526 DOI: 10.1152/Ajpcell.00456.2008 |
0.446 |
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| 2009 |
Hilario JD, Rodino-Klapac LR, Wang C, Beattie CE. Semaphorin 5A is a bifunctional axon guidance cue for axial motoneurons in vivo. Developmental Biology. 326: 190-200. PMID 19059233 DOI: 10.1016/J.Ydbio.2008.11.007 |
0.443 |
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| 2008 |
Rodino-Klapac LR, Lee JS, Mulligan RC, Clark KR, Mendell JR. Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D. Neurology. 71: 240-7. PMID 18525034 DOI: 10.1212/01.Wnl.0000306309.85301.E2 |
0.425 |
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| 2007 |
Rodino-Klapac LR, Janssen PM, Montgomery CL, Coley BD, Chicoine LG, Clark KR, Mendell JR. A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy. Journal of Translational Medicine. 5: 45. PMID 17892583 DOI: 10.1186/1479-5876-5-45 |
0.491 |
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| 2007 |
Rodino-Klapac LR, Chicoine LG, Kaspar BK, Mendell JR. Gene therapy for duchenne muscular dystrophy: expectations and challenges. Archives of Neurology. 64: 1236-41. PMID 17846262 DOI: 10.1001/Archneur.64.9.1236 |
0.489 |
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| 2004 |
Rodino-Klapac LR, Beattie CE. Zebrafish topped is required for ventral motor axon guidance. Developmental Biology. 273: 308-20. PMID 15328015 DOI: 10.1016/J.Ydbio.2004.06.007 |
0.473 |
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