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Items: 46

1.

An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque.

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, Chicoine LG, Martin PT.

Mol Ther Methods Clin Dev. 2018 Jul 14;10:89-104. doi: 10.1016/j.omtm.2018.06.002. eCollection 2018 Sep 21.

2.

Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production.

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.

Neurology. 2018 Jun 12;90(24):e2146-e2154. doi: 10.1212/WNL.0000000000005680. Epub 2018 May 11. Erratum in: Neurology. 2018 Sep 25;91(13):637.

PMID:
29752304
3.

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.

Wallace LM, Saad NY, Pyne NK, Fowler AM, Eidahl JO, Domire JS, Griffin DA, Herman AC, Sahenk Z, Rodino-Klapac LR, Harper SQ.

Mol Ther Methods Clin Dev. 2017 Dec 24;8:121-130. doi: 10.1016/j.omtm.2017.12.005. eCollection 2018 Mar 16.

4.

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

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, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK.

N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198.

5.

Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.

Potter RA, Griffin DA, Sondergaard PC, Johnson RW, Pozsgai ER, Heller KN, Peterson EL, Lehtimäki KK, Windish HP, Mittal PJ, Albrecht DE, Mendell JR, Rodino-Klapac LR.

Hum Gene Ther. 2018 Jul;29(7):749-762. doi: 10.1089/hum.2017.062. Epub 2017 Jul 13.

6.

MicroRNA-29 overexpression by adeno-associated virus suppresses fibrosis and restores muscle function in combination with micro-dystrophin.

Heller KN, Mendell JT, Mendell JR, Rodino-Klapac LR.

JCI Insight. 2017 May 4;2(9). pii: 93309. doi: 10.1172/jci.insight.93309. [Epub ahead of print]

7.

Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone.

Cramer ML, Shao G, Rodino-Klapac LR, Chicoine LG, Martin PT.

Hum Gene Ther. 2017 Jun;28(6):493-509. doi: 10.1089/hum.2016.113. Epub 2017 Mar 23.

8.

Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice.

Pozsgai ER, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR.

Mol Ther. 2017 Apr 5;25(4):855-869. doi: 10.1016/j.ymthe.2017.02.013. Epub 2017 Mar 9.

9.

Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.

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, Lewis S, McColly M, Hogan MJ, Kaspar BK.

Mol Ther. 2017 Apr 5;25(4):870-879. doi: 10.1016/j.ymthe.2017.02.015. Epub 2017 Mar 6.

10.

Duchenne muscular dystrophy: CRISPR/Cas9 treatment.

Mendell JR, Rodino-Klapac LR.

Cell Res. 2016 May;26(5):513-4. doi: 10.1038/cr.2016.28. Epub 2016 Mar 1.

11.

Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy.

Griffin DA, Johnson RW, Whitlock JM, Pozsgai ER, Heller KN, Grose WE, Arnold WD, Sahenk Z, Hartzell HC, Rodino-Klapac LR.

Hum Mol Genet. 2016 May 15;25(10):1900-1911. Epub 2016 Feb 23.

12.

Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy.

Al-Zaidy SA, Sahenk Z, Rodino-Klapac LR, Kaspar B, Mendell JR.

J Neuromuscul Dis. 2015 Sep 2;2(3):185-192.

13.

β-Sarcoglycan gene transfer decreases fibrosis and restores force in LGMD2E mice.

Pozsgai ER, Griffin DA, Heller KN, Mendell JR, Rodino-Klapac LR.

Gene Ther. 2016 Jan;23(1):57-66. doi: 10.1038/gt.2015.80. Epub 2015 Aug 20.

PMID:
26214262
14.

Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice.

Heller KN, Montgomery CL, Shontz KM, Clark KR, Mendell JR, Rodino-Klapac LR.

Hum Gene Ther. 2015 Oct;26(10):647-56. doi: 10.1089/hum.2015.062. Epub 2015 Aug 11.

15.

Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

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, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2015 May;21(5):537. doi: 10.1038/nm0515-537c. No abstract available.

PMID:
25951531
16.

Corrigendum: Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

Wein N, Vulin A, Falzarano MS, Szigyarto CA, Maiti B, Findlay A, Heller KN, Uhlén M, Bakthavachalu B, Messina S, Vita G, Passarelli C, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2015 Apr;21(4):414. doi: 10.1038/nm0415-414b. No abstract available.

PMID:
25849273
17.

AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models.

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.

Ann Clin Transl Neurol. 2015 Mar;2(3):256-70. doi: 10.1002/acn3.172. Epub 2015 Jan 20.

18.

A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.

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, Al-Zaidy S, Govoni A, Rodino-Klapac LR, Hogan MJ, Kaspar BK.

Mol Ther. 2015 Jan;23(1):192-201. doi: 10.1038/mt.2014.200. Epub 2014 Oct 17.

19.

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice.

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, Gualandi F, Wilton SD, Rodino-Klapac LR, Yang L, Dunn DM, Schoenberg DR, Weiss RB, Howard MT, Ferlini A, Flanigan KM.

Nat Med. 2014 Sep;20(9):992-1000. doi: 10.1038/nm.3628. Epub 2014 Aug 10. Erratum in: Nat Med. 2015 Apr;21(4):414. Erratum in: Nat Med. 2015 May;21(5):537. Brioschi, Simona [added]; Bovolenta, Matteo [added]; Neri, Marcella [added].

20.

Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.

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, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR.

Mol Ther. 2014 Feb;22(2):338-347. doi: 10.1038/mt.2013.244. Epub 2013 Oct 23.

21.

AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy.

Sahenk Z, Galloway G, Clark KR, Malik V, Rodino-Klapac LR, Kaspar BK, Chen L, Braganza C, Montgomery C, Mendell JR.

Mol Ther. 2014 Mar;22(3):511-521. doi: 10.1038/mt.2013.250. Epub 2013 Oct 28.

22.

Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.

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, Sahenk Z, Mendell JR, Martin PT.

Mol Ther. 2014 Apr;22(4):713-24. doi: 10.1038/mt.2013.246. Epub 2013 Oct 22.

23.

MicroRNA based treatment of cardiomyopathy: not all dystrophies are created equal.

Rodino-Klapac LR.

J Am Heart Assoc. 2013 Aug 22;2(4):e000384. doi: 10.1161/JAHA.113.000384. No abstract available.

24.

Eteplirsen for the treatment of Duchenne muscular dystrophy.

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, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM; Eteplirsen Study Group.

Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10.

25.

Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.

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.

Hum Mol Genet. 2013 Dec 15;22(24):4929-37. doi: 10.1093/hmg/ddt342. Epub 2013 Jul 17.

26.

Impaired regeneration in LGMD2A supported by increased PAX7-positive satellite cell content and muscle-specific microrna dysregulation.

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.

Muscle Nerve. 2013 May;47(5):731-9. doi: 10.1002/mus.23669. Epub 2013 Mar 29.

27.

Update on the treatment of Duchenne muscular dystrophy.

Rodino-Klapac LR, Mendell JR, Sahenk Z.

Curr Neurol Neurosci Rep. 2013 Mar;13(3):332. doi: 10.1007/s11910-012-0332-1. Review.

28.

AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice.

Heller KN, Montgomery CL, Janssen PM, Clark KR, Mendell JR, Rodino-Klapac LR.

Mol Ther. 2013 Mar;21(3):520-5. doi: 10.1038/mt.2012.281. Epub 2013 Jan 15.

29.

Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer.

Grose WE, Clark KR, Griffin D, Malik V, Shontz KM, Montgomery CL, Lewis S, Brown RH Jr, Janssen PM, Mendell JR, Rodino-Klapac LR.

PLoS One. 2012;7(6):e39233. doi: 10.1371/journal.pone.0039233. Epub 2012 Jun 15.

30.

Emerging drugs for Duchenne muscular dystrophy.

Malik V, Rodino-Klapac LR, Mendell JR.

Expert Opin Emerg Drugs. 2012 Jun;17(2):261-77. doi: 10.1517/14728214.2012.691965. Review.

31.

mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx Mice.

Beastrom N, Lu H, Macke A, Canan BD, Johnson EK, Penton CM, Kaspar BK, Rodino-Klapac LR, Zhou L, Janssen PM, Montanaro F.

Am J Pathol. 2011 Nov;179(5):2464-74. doi: 10.1016/j.ajpath.2011.07.009. Epub 2011 Sep 3.

32.

AAV-mediated gene therapy to the isolated limb in rhesus macaques.

Rodino-Klapac LR, Montgomery CL, Mendell JR, Chicoine LG.

Methods Mol Biol. 2011;709:287-98. doi: 10.1007/978-1-61737-982-6_19.

PMID:
21194036
33.

Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy.

Malik V, Rodino-Klapac LR, Viollet L, Mendell JR.

Ther Adv Neurol Disord. 2010 Nov;3(6):379-89. doi: 10.1177/1756285610388693.

34.

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.

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, Astbury C, Kota J, Sahenk Z, Walker CM, Clark KR.

Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251.

35.

Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy.

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, Watts V, Sivakumar K, Bien-Willner R, Flanigan KM, Sahenk Z, Barohn RJ, Walker CM, Mendell JR.

Ann Neurol. 2010 Jun;67(6):771-80. doi: 10.1002/ana.22024.

PMID:
20517938
36.

Molecular therapeutic strategies targeting Duchenne muscular dystrophy.

Mendell JR, Rodino-Klapac LR, Malik V.

J Child Neurol. 2010 Sep;25(9):1145-8. doi: 10.1177/0883073810371005. Epub 2010 May 24. Review.

37.

Follistatin gene delivery enhances muscle growth and strength in nonhuman primates.

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, Kaspar BK.

Sci Transl Med. 2009 Nov 11;1(6):6ra15. doi: 10.1126/scitranslmed.3000112.

38.

Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery.

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.

Mol Ther. 2010 Jan;18(1):109-17. doi: 10.1038/mt.2009.254. Epub 2009 Nov 10.

39.

Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.

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, Walker CM, Sahenk Z, Clark KR.

Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732.

40.

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease.

Rodino-Klapac LR, Haidet AM, Kota J, Handy C, Kaspar BK, Mendell JR.

Muscle Nerve. 2009 Mar;39(3):283-96. doi: 10.1002/mus.21244. Review.

41.

Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice.

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.

Am J Physiol Cell Physiol. 2009 Mar;296(3):C476-88. doi: 10.1152/ajpcell.00456.2008. Epub 2008 Dec 24.

42.

Semaphorin 5A is a bifunctional axon guidance cue for axial motoneurons in vivo.

Hilario JD, Rodino-Klapac LR, Wang C, Beattie CE.

Dev Biol. 2009 Feb 1;326(1):190-200. doi: 10.1016/j.ydbio.2008.11.007. Epub 2008 Nov 20.

43.

Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

Rodino-Klapac LR, Lee JS, Mulligan RC, Clark KR, Mendell JR.

Neurology. 2008 Jul 22;71(4):240-7. doi: 10.1212/01.wnl.0000306309.85301.e2. Epub 2008 Jun 4.

PMID:
18525034
44.

A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy.

Rodino-Klapac LR, Janssen PM, Montgomery CL, Coley BD, Chicoine LG, Clark KR, Mendell JR.

J Transl Med. 2007 Sep 24;5:45.

45.

Gene therapy for duchenne muscular dystrophy: expectations and challenges.

Rodino-Klapac LR, Chicoine LG, Kaspar BK, Mendell JR.

Arch Neurol. 2007 Sep;64(9):1236-41. Review.

PMID:
17846262
46.

Zebrafish topped is required for ventral motor axon guidance.

Rodino-Klapac LR, Beattie CE.

Dev Biol. 2004 Sep 15;273(2):308-20.

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