Format
Sort by
Items per page

Send to

Choose Destination

Links from PubMed

Items: 1 to 20 of 111

1.

MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy.

Barbash IM, Cecchini S, Faranesh AZ, Virag T, Li L, Yang Y, Hoyt RF, Kornegay JN, Bogan JR, Garcia L, Lederman RJ, Kotin RM.

Gene Ther. 2013 Mar;20(3):274-82. doi: 10.1038/gt.2012.38. Epub 2012 May 3.

2.

Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping.

Bish LT, Sleeper MM, Forbes SC, Wang B, Reynolds C, Singletary GE, Trafny D, Morine KJ, Sanmiguel J, Cecchini S, Virag T, Vulin A, Beley C, Bogan J, Wilson JM, Vandenborne K, Kornegay JN, Walter GA, Kotin RM, Garcia L, Sweeney HL.

Mol Ther. 2012 Mar;20(3):580-9. doi: 10.1038/mt.2011.264. Epub 2011 Dec 6.

3.

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.

4.

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.

PMID:
21194028
5.

Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

Le Guiner C, Montus M, Servais L, Cherel Y, Francois V, Thibaud JL, Wary C, Matot B, Larcher T, Guigand L, Dutilleul M, Domenger C, Allais M, Beuvin M, Moraux A, Le Duff J, Devaux M, Jaulin N, Guilbaud M, Latournerie V, Veron P, Boutin S, Leborgne C, Desgue D, Deschamps JY, Moullec S, Fromes Y, Vulin A, Smith RH, Laroudie N, Barnay-Toutain F, Rivière C, Bucher S, Le TH, Delaunay N, Gasmi M, Kotin RM, Bonne G, Adjali O, Masurier C, Hogrel JY, Carlier P, Moullier P, Voit T.

Mol Ther. 2014 Nov;22(11):1923-35. doi: 10.1038/mt.2014.151. Epub 2014 Aug 4.

6.

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.

PMID:
16724091
7.

In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.

El Refaey M, Xu L, Gao Y, Canan BD, Adesanya TMA, Warner SC, Akagi K, Symer DE, Mohler PJ, Ma J, Janssen PML, Han R.

Circ Res. 2017 Sep 29;121(8):923-929. doi: 10.1161/CIRCRESAHA.117.310996. Epub 2017 Aug 8.

PMID:
28790199
8.

A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping.

Walmsley GL, Arechavala-Gomeza V, Fernandez-Fuente M, Burke MM, Nagel N, Holder A, Stanley R, Chandler K, Marks SL, Muntoni F, Shelton GD, Piercy RJ.

PLoS One. 2010 Jan 13;5(1):e8647. doi: 10.1371/journal.pone.0008647.

9.

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.

10.

In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.

Wang Q, Yin H, Camelliti P, Betts C, Moulton H, Lee H, Saleh AF, Gait MJ, Wood MJ.

J Gene Med. 2010 Apr;12(4):354-64. doi: 10.1002/jgm.1446.

PMID:
20235089
11.

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.

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

Hum Mol Genet. 2012 Jun 1;21(11):2559-71. doi: 10.1093/hmg/dds082. Epub 2012 Mar 2.

12.

Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice.

Denti MA, Rosa A, D'Antona G, Sthandier O, De Angelis FG, Nicoletti C, Allocca M, Pansarasa O, Parente V, Musarò A, Auricchio A, Bottinelli R, Bozzoni I.

Hum Gene Ther. 2006 May;17(5):565-74.

PMID:
16716113
13.

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.

Yue Y, Pan X, Hakim CH, Kodippili K, Zhang K, Shin JH, Yang HT, McDonald T, Duan D.

Hum Mol Genet. 2015 Oct 15;24(20):5880-90. doi: 10.1093/hmg/ddv310. Epub 2015 Aug 11.

14.

Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products.

Yuasa K, Yoshimura M, Urasawa N, Ohshima S, Howell JM, Nakamura A, Hijikata T, Miyagoe-Suzuki Y, Takeda S.

Gene Ther. 2007 Sep;14(17):1249-60. Epub 2007 Jun 21.

PMID:
17581597
15.

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.

Le Hir M, Goyenvalle A, Peccate C, Précigout G, Davies KE, Voit T, Garcia L, Lorain S.

Mol Ther. 2013 Aug;21(8):1551-8. doi: 10.1038/mt.2013.121. Epub 2013 Jun 11.

16.

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.

17.

Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.

Denti MA, Rosa A, D'Antona G, Sthandier O, De Angelis FG, Nicoletti C, Allocca M, Pansarasa O, Parente V, Musarò A, Auricchio A, Bottinelli R, Bozzoni I.

Proc Natl Acad Sci U S A. 2006 Mar 7;103(10):3758-63. Epub 2006 Feb 24.

18.

Chimeric RNA/ethylene-bridged nucleic acids promote dystrophin expression in myocytes of duchenne muscular dystrophy by inducing skipping of the nonsense mutation-encoding exon.

Surono A, Van Khanh T, Takeshima Y, Wada H, Yagi M, Takagi M, Koizumi M, Matsuo M.

Hum Gene Ther. 2004 Aug;15(8):749-57.

PMID:
15319032
19.

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.

20.

Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice.

Koo T, Popplewell L, Athanasopoulos T, Dickson G.

Hum Gene Ther. 2014 Feb;25(2):98-108. doi: 10.1089/hum.2013.164. Epub 2013 Dec 19.

PMID:
24191945

Supplemental Content

Support Center