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Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts.

Cazzella V, Martone J, Pinnarò C, Santini T, Twayana SS, Sthandier O, D'Amico A, Ricotti V, Bertini E, Muntoni F, Bozzoni I.

Mol Ther. 2012 Nov;20(11):2134-42. doi: 10.1038/mt.2012.178. Epub 2012 Sep 11.


Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping.

Incitti T, De Angelis FG, Cazzella V, Sthandier O, Pinnarò C, Legnini I, Bozzoni I.

Mol Ther. 2010 Sep;18(9):1675-82. doi: 10.1038/mt.2010.123. Epub 2010 Jun 15.


U1 snRNA as an effective vector for stable expression of antisense molecules and for the inhibition of the splicing reaction.

Martone J, De Angelis FG, Bozzoni I.

Methods Mol Biol. 2012;867:239-57. doi: 10.1007/978-1-61779-767-5_16.


Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells.

De Angelis FG, Sthandier O, Berarducci B, Toso S, Galluzzi G, Ricci E, Cossu G, Bozzoni I.

Proc Natl Acad Sci U S A. 2002 Jul 9;99(14):9456-61. Epub 2002 Jun 20.


Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.

Goyenvalle A, Davies KE.

Methods Mol Biol. 2011;709:179-96. doi: 10.1007/978-1-61737-982-6_11.


Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials.

Anthony K, Cirak S, Torelli S, Tasca G, Feng L, Arechavala-Gomeza V, Armaroli A, Guglieri M, Straathof CS, Verschuuren JJ, Aartsma-Rus A, Helderman-van den Enden P, Bushby K, Straub V, Sewry C, Ferlini A, Ricci E, Morgan JE, Muntoni F.

Brain. 2011 Dec;134(Pt 12):3547-59. doi: 10.1093/brain/awr291. Epub 2011 Nov 18. Erratum in: Brain. 2016 Apr;139(Pt 4):e27.


Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.

Vulin A, Barthélémy I, Goyenvalle A, Thibaud JL, Beley C, Griffith G, Benchaouir R, le Hir M, Unterfinger Y, Lorain S, Dreyfus P, Voit T, Carlier P, Blot S, Garcia L.

Mol Ther. 2012 Nov;20(11):2120-33. doi: 10.1038/mt.2012.181. Epub 2012 Sep 11.


Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.

Anthony K, Arechavala-Gomeza V, Ricotti V, Torelli S, Feng L, Janghra N, Tasca G, Guglieri M, Barresi R, Armaroli A, Ferlini A, Bushby K, Straub V, Ricci E, Sewry C, Morgan J, Muntoni F.

JAMA Neurol. 2014 Jan;71(1):32-40. doi: 10.1001/jamaneurol.2013.4908.


Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.

Goyenvalle A, Wright J, Babbs A, Wilkins V, Garcia L, Davies KE.

Mol Ther. 2012 Jun;20(6):1212-21. doi: 10.1038/mt.2012.26. Epub 2012 Feb 21.


Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse.

Mann CJ, Honeyman K, Cheng AJ, Ly T, Lloyd F, Fletcher S, Morgan JE, Partridge TA, Wilton SD.

Proc Natl Acad Sci U S A. 2001 Jan 2;98(1):42-7.


Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy.

Cirak S, Feng L, Anthony K, Arechavala-Gomeza V, Torelli S, Sewry C, Morgan JE, Muntoni F.

Mol Ther. 2012 Feb;20(2):462-7. doi: 10.1038/mt.2011.248. Epub 2011 Nov 15.


Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.

Goyenvalle A, Babbs A, van Ommen GJ, Garcia L, Davies KE.

Mol Ther. 2009 Jul;17(7):1234-40. doi: 10.1038/mt.2009.113. Epub 2009 May 19.


Variable phenotype of del45-55 Becker patients correlated with nNOSμ mislocalization and RYR1 hypernitrosylation.

Gentil C, Leturcq F, Ben Yaou R, Kaplan JC, Laforet P, Pénisson-Besnier I, Espil-Taris C, Voit T, Garcia L, Piétri-Rouxel F.

Hum Mol Genet. 2012 Aug 1;21(15):3449-60. doi: 10.1093/hmg/dds176. Epub 2012 May 15.


Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.

Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O.

Science. 2004 Dec 3;306(5702):1796-9. Epub 2004 Nov 4.


Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, den Dunnen JT, Baas F, van Ommen GJ, van Deutekom JC.

Hum Mol Genet. 2003 Apr 15;12(8):907-14.


Engineering U7snRNA gene to reframe transcripts.

Goyenvalle A.

Methods Mol Biol. 2012;867:259-71. doi: 10.1007/978-1-61779-767-5_17.


Absence of neuronal nitric oxide synthase (nNOS) as a pathological marker for the diagnosis of Becker muscular dystrophy with rod domain deletions.

Torelli S, Brown SC, Jimenez-Mallebrera C, Feng L, Muntoni F, Sewry CA.

Neuropathol Appl Neurobiol. 2004 Oct;30(5):540-5.


miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy.

Cacchiarelli D, Incitti T, Martone J, Cesana M, Cazzella V, Santini T, Sthandier O, Bozzoni I.

EMBO Rep. 2011 Feb;12(2):136-41. doi: 10.1038/embor.2010.208. Epub 2011 Jan 7.


Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy.

Meng J, Counsell JR, Reza M, Laval SH, Danos O, Thrasher A, Lochmüller H, Muntoni F, Morgan JE.

Sci Rep. 2016 Jan 27;6:19750. doi: 10.1038/srep19750.


Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD.

McClorey G, Moulton HM, Iversen PL, Fletcher S, Wilton SD.

Gene Ther. 2006 Oct;13(19):1373-81. Epub 2006 May 25.


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