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Items: 1 to 20 of 102


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.


Combined effect of AAV-U7-induced dystrophin exon skipping and soluble activin Type IIB receptor in mdx mice.

Hoogaars WM, Mouisel E, Pasternack A, Hulmi JJ, Relizani K, Schuelke M, Schirwis E, Garcia L, Ritvos O, Ferry A, 't Hoen PA, Amthor H.

Hum Gene Ther. 2012 Dec;23(12):1269-79. doi: 10.1089/hum.2012.056.


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.


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.


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.


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.


Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice.

Benchaouir R, Meregalli M, Farini A, D'Antona G, Belicchi M, Goyenvalle A, Battistelli M, Bresolin N, Bottinelli R, Garcia L, Torrente Y.

Cell Stem Cell. 2007 Dec 13;1(6):646-57. doi: 10.1016/j.stem.2007.09.016.


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.


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.


Restoration of all dystrophin protein interactions by functional domains in trans does not rescue dystrophy.

Gardner KL, Kearney JA, Edwards JD, Rafael-Fortney JA.

Gene Ther. 2006 May;13(9):744-51.


Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice.

Denti MA, Incitti T, Sthandier O, Nicoletti C, De Angelis FG, Rizzuto E, Auricchio A, Musarò A, Bozzoni I.

Hum Gene Ther. 2008 Jun;19(6):601-8. doi: 10.1089/hum.2008.012.


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.


Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy.

Zhang Y, Yue Y, Li L, Hakim CH, Zhang K, Thomas GD, Duan D.

Hum Mol Genet. 2013 Sep 15;22(18):3720-9. doi: 10.1093/hmg/ddt224.


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.


A canine minidystrophin is functional and therapeutic in mdx mice.

Wang B, Li J, Qiao C, Chen C, Hu P, Zhu X, Zhou L, Bogan J, Kornegay J, Xiao X.

Gene Ther. 2008 Aug;15(15):1099-106. doi: 10.1038/gt.2008.70.


Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene.

Koppanati BM, Li J, Reay DP, Wang B, Daood M, Zheng H, Xiao X, Watchko JF, Clemens PR.

Gene Ther. 2010 Nov;17(11):1355-62. doi: 10.1038/gt.2010.84.


Autologous transplantation of muscle precursor cells modified with a lentivirus for muscular dystrophy: human cells and primate models.

Quenneville SP, Chapdelaine P, Skuk D, Paradis M, Goulet M, Rousseau J, Xiao X, Garcia L, Tremblay JP.

Mol Ther. 2007 Feb;15(2):431-8.


Multi-parametric MRI at 14T for muscular dystrophy mice treated with AAV vector-mediated gene therapy.

Park J, Wicki J, Knoblaugh SE, Chamberlain JS, Lee D.

PLoS One. 2015 Apr 9;10(4):e0124914. doi: 10.1371/journal.pone.0124914.


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.

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