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Results: 1 to 20 of 498

1.

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.

PMID:
15528407
[PubMed - indexed for MEDLINE]
Free Article
2.

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.

Lu QL, Mann CJ, Lou F, Bou-Gharios G, Morris GE, Xue SA, Fletcher S, Partridge TA, Wilton SD.

Nat Med. 2003 Aug;9(8):1009-14. Epub 2003 Jul 6.

PMID:
12847521
[PubMed - indexed for MEDLINE]
3.

U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping.

Brun C, Suter D, Pauli C, Dunant P, Lochmüller H, Burgunder JM, Schümperli D, Weis J.

Cell Mol Life Sci. 2003 Mar;60(3):557-66.

PMID:
12737315
[PubMed - indexed for MEDLINE]
4.
5.

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.

PMID:
23752313
[PubMed - indexed for MEDLINE]
6.

Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy.

Ahmad A, Brinson M, Hodges BL, Chamberlain JS, Amalfitano A.

Hum Mol Genet. 2000 Oct 12;9(17):2507-15.

PMID:
11030755
[PubMed - indexed for MEDLINE]
Free Article
7.

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.

PMID:
22968479
[PubMed - indexed for MEDLINE]
Free PMC Article
8.
9.

Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides.

Wilton SD, Lloyd F, Carville K, Fletcher S, Honeyman K, Agrawal S, Kole R.

Neuromuscul Disord. 1999 Jul;9(5):330-8.

PMID:
10407856
[PubMed - indexed for MEDLINE]
10.

Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers.

Williams JH, Sirsi SR, Latta DR, Lutz GJ.

Mol Ther. 2006 Jul;14(1):88-96. Epub 2006 Feb 20.

PMID:
16488666
[PubMed - indexed for MEDLINE]
11.

Potential of oligonucleotide-mediated exon-skipping therapy for Duchenne muscular dystrophy.

Yokota T, Pistilli E, Duddy W, Nagaraju K.

Expert Opin Biol Ther. 2007 Jun;7(6):831-42. Review.

PMID:
17555369
[PubMed - indexed for MEDLINE]
12.

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.

PMID:
16501048
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Screening for antisense modulation of dystrophin pre-mRNA splicing.

Dickson G, Hill V, Graham IR.

Neuromuscul Disord. 2002 Oct;12 Suppl 1:S67-70. Review.

PMID:
12206799
[PubMed - indexed for MEDLINE]
14.

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.

PMID:
18371406
[PubMed - indexed for MEDLINE]
Free Article
15.

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
[PubMed - indexed for MEDLINE]
16.

Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy.

Kendall GC, Mokhonova EI, Moran M, Sejbuk NE, Wang DW, Silva O, Wang RT, Martinez L, Lu QL, Damoiseaux R, Spencer MJ, Nelson SF, Miceli MC.

Sci Transl Med. 2012 Dec 12;4(164):164ra160. doi: 10.1126/scitranslmed.3005054.

PMID:
23241744
[PubMed - indexed for MEDLINE]
Free Article
17.

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
[PubMed - indexed for MEDLINE]
18.

Induced dystrophin exon skipping in human muscle explants.

McClorey G, Fall AM, Moulton HM, Iversen PL, Rasko JE, Ryan M, Fletcher S, Wilton SD.

Neuromuscul Disord. 2006 Oct;16(9-10):583-90. Epub 2006 Aug 21.

PMID:
16919955
[PubMed - indexed for MEDLINE]
19.

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology.

Alter J, Lou F, Rabinowitz A, Yin H, Rosenfeld J, Wilton SD, Partridge TA, Lu QL.

Nat Med. 2006 Feb;12(2):175-7. Epub 2006 Jan 29.

PMID:
16444267
[PubMed - indexed for MEDLINE]
20.

Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle.

Wells KE, Fletcher S, Mann CJ, Wilton SD, Wells DJ.

FEBS Lett. 2003 Sep 25;552(2-3):145-9.

PMID:
14527677
[PubMed - indexed for MEDLINE]
Free Article

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