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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]


Progress toward Gene Therapy for Duchenne Muscular Dystrophy.

Chamberlain JR, Chamberlain JS.

Mol Ther. 2017 May 3;25(5):1125-1131. doi: 10.1016/j.ymthe.2017.02.019. Epub 2017 Apr 15. Review.


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.


Systemic delivery of adeno-associated viral vectors.

Duan D.

Curr Opin Virol. 2016 Dec;21:16-25. doi: 10.1016/j.coviro.2016.07.006. Epub 2016 Jul 25. Review.


piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts.

Loperfido M, Jarmin S, Dastidar S, Di Matteo M, Perini I, Moore M, Nair N, Samara-Kuko E, Athanasopoulos T, Tedesco FS, Dickson G, Sampaolesi M, VandenDriessche T, Chuah MK.

Nucleic Acids Res. 2016 Jan 29;44(2):744-60. doi: 10.1093/nar/gkv1464. Epub 2015 Dec 17.


Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.

Gruntman AM, Flotte TR.

Hum Gene Ther Clin Dev. 2015 Sep;26(3):159-64. doi: 10.1089/humc.2015.116. Epub 2015 Sep 10. Review.


Robust Lentiviral Gene Delivery But Limited Transduction Capacity of Commonly Used Adeno-Associated Viral Serotypes in Xenotransplanted Human Skin.

Jakobsen M, Askou AL, Stenderup K, Rosada C, Dagnæs-Hansen F, Jensen TG, Corydon TJ, Mikkelsen JG, Aagaard L.

Hum Gene Ther Methods. 2015 Aug;26(4):123-33. doi: 10.1089/hgtb.2014.135.


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.


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.


Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice.

Betts CA, Saleh AF, Carr CA, Hammond SM, Coenen-Stass AM, Godfrey C, McClorey G, Varela MA, Roberts TC, Clarke K, Gait MJ, Wood MJ.

Sci Rep. 2015 Mar 11;5:8986. doi: 10.1038/srep08986.


Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice.

Duncan FJ, Naughton BJ, Zaraspe K, Murrey DA, Meadows AS, Clark KR, Newsom DE, White P, Fu H, McCarty DM.

Mol Ther. 2015 Apr;23(4):638-47. doi: 10.1038/mt.2015.9. Epub 2015 Jan 16.


Gene therapy for muscular dystrophy: moving the field forward.

Al-Zaidy S, Rodino-Klapac L, Mendell JR.

Pediatr Neurol. 2014 Nov;51(5):607-18. doi: 10.1016/j.pediatrneurol.2014.08.002. Epub 2014 Aug 7. Review.


Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting.

Swiderski K, Shaffer SA, Gallis B, Odom GL, Arnett AL, Scott Edgar J, Baum DM, Chee A, Naim T, Gregorevic P, Murphy KT, Moody J, Goodlett DR, Lynch GS, Chamberlain JS.

Hum Mol Genet. 2014 Dec 20;23(25):6697-711. doi: 10.1093/hmg/ddu388. Epub 2014 Jul 31.


Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes.

Larsen CA, Howard MT.

Neuromuscul Disord. 2014 Aug;24(8):693-706. doi: 10.1016/j.nmd.2014.05.006. Epub 2014 May 22.


Therapy of Genetic Disorders-Novel Therapies for Duchenne Muscular Dystrophy.

Seto JT, Bengtsson NE, Chamberlain JS.

Curr Pediatr Rep. 2014 Jun 1;2(2):102-112.


Novel adeno-associated viral vector delivering the utrophin gene regulator jazz counteracts dystrophic pathology in mdx mice.

Strimpakos G, Corbi N, Pisani C, Di Certo MG, Onori A, Luvisetto S, Severini C, Gabanella F, Monaco L, Mattei E, Passananti C.

J Cell Physiol. 2014 Sep;229(9):1283-91. doi: 10.1002/jcp.24567.


The potential of adeno-associated viral vectors for gene delivery to muscle tissue.

Wang D, Zhong L, Nahid MA, Gao G.

Expert Opin Drug Deliv. 2014 Mar;11(3):345-364. doi: 10.1517/17425247.2014.871258. Epub 2014 Jan 3. Review.


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):338-47. doi: 10.1038/mt.2013.244. Epub 2013 Oct 23.


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.

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