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Items: 1 to 20 of 105

1.

rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

Gregorevic P, Allen JM, Minami E, Blankinship MJ, Haraguchi M, Meuse L, Finn E, Adams ME, Froehner SC, Murry CE, Chamberlain JS.

Nat Med. 2006 Jul;12(7):787-9. Epub 2006 Jul 2.

2.

Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.

Koo T, Malerba A, Athanasopoulos T, Trollet C, Boldrin L, Ferry A, Popplewell L, Foster H, Foster K, Dickson G.

Hum Gene Ther. 2011 Nov;22(11):1379-88. doi: 10.1089/hum.2011.020. Epub 2011 May 25.

3.

Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice.

Odom GL, Gregorevic P, Allen JM, Finn E, Chamberlain JS.

Mol Ther. 2008 Sep;16(9):1539-45. doi: 10.1038/mt.2008.149. Epub 2008 Jul 29.

5.

Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer.

Foster H, Sharp PS, Athanasopoulos T, Trollet C, Graham IR, Foster K, Wells DJ, Dickson G.

Mol Ther. 2008 Nov;16(11):1825-32. doi: 10.1038/mt.2008.186. Epub 2008 Sep 2.

6.

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
7.

Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance.

Hayashita-Kinoh H, Yugeta N, Okada H, Nitahara-Kasahara Y, Chiyo T, Okada T, Takeda S.

Mol Ther. 2015 Apr;23(4):627-37. doi: 10.1038/mt.2015.5. Epub 2015 Jan 14.

8.

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.

9.

rAAV6-microdystrophin rescues aberrant Golgi complex organization in mdx skeletal muscles.

Percival JM, Gregorevic P, Odom GL, Banks GB, Chamberlain JS, Froehner SC.

Traffic. 2007 Oct;8(10):1424-39. Epub 2007 Aug 20.

10.

Herpes simplex virus VP22 enhances adenovirus-mediated microdystrophin gene transfer to skeletal muscles in dystrophin-deficient (mdx) mice.

Xiong F, Xiao S, Peng F, Zheng H, Yu M, Ruan Y, Li W, Shang Y, Zhao C, Zhou W, Chen H, Chamberlain JS, Fang L, Zhang C.

Hum Gene Ther. 2007 Jun;18(6):490-501.

PMID:
17550336
11.

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
12.

Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.

Gregorevic P, Blankinship MJ, Allen JM, Chamberlain JS.

Mol Ther. 2008 Apr;16(4):657-64. doi: 10.1038/mt.2008.28. Epub 2008 Mar 4.

13.

Gene-mediated restoration of normal myofiber elasticity in dystrophic muscles.

Puttini S, Lekka M, Dorchies OM, Saugy D, Incitti T, Ruegg UT, Bozzoni I, Kulik AJ, Mermod N.

Mol Ther. 2009 Jan;17(1):19-25. doi: 10.1038/mt.2008.239. Epub 2008 Nov 11.

14.

Phenotypic improvement of dystrophic muscles by rAAV/microdystrophin vectors is augmented by Igf1 codelivery.

Abmayr S, Gregorevic P, Allen JM, Chamberlain JS.

Mol Ther. 2005 Sep;12(3):441-50.

15.

Enhanced expression of the alpha 7 beta 1 integrin reduces muscular dystrophy and restores viability in dystrophic mice.

Burkin DJ, Wallace GQ, Nicol KJ, Kaufman DJ, Kaufman SJ.

J Cell Biol. 2001 Mar 19;152(6):1207-18.

16.

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.

17.

AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype.

Yoshimura M, Sakamoto M, Ikemoto M, Mochizuki Y, Yuasa K, Miyagoe-Suzuki Y, Takeda S.

Mol Ther. 2004 Nov;10(5):821-8.

18.

Functional protection of dystrophic mouse (mdx) muscles after adenovirus-mediated transfer of a dystrophin minigene.

Deconinck N, Ragot T, Maréchal G, Perricaudet M, Gillis JM.

Proc Natl Acad Sci U S A. 1996 Apr 16;93(8):3570-4.

19.

Codon optimization of the microdystrophin gene for Duchene muscular dystrophy gene therapy.

Athanasopoulos T, Foster H, Foster K, Dickson G.

Methods Mol Biol. 2011;709:21-37. doi: 10.1007/978-1-61737-982-6_2.

PMID:
21194019
20.

Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD).

Athanasopoulos T, Graham IR, Foster H, Dickson G.

Gene Ther. 2004 Oct;11 Suppl 1:S109-21. Review.

PMID:
15454965

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