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Items: 1 to 20 of 294

1.

Peptide-morpholino conjugate: a promising therapeutic for Duchenne muscular dystrophy.

Moulton HM, Wu B, Jearawiriyapaisarn N, Sazani P, Lu QL, Kole R.

Ann N Y Acad Sci. 2009 Sep;1175:55-60. doi: 10.1111/j.1749-6632.2009.04976.x.

PMID:
19796077
2.

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer.

Wu B, Moulton HM, Iversen PL, Jiang J, Li J, Li J, Spurney CF, Sali A, Guerron AD, Nagaraju K, Doran T, Lu P, Xiao X, Lu QL.

Proc Natl Acad Sci U S A. 2008 Sep 30;105(39):14814-9. doi: 10.1073/pnas.0805676105. Epub 2008 Sep 19.

3.

By-passing the nonsense mutation in the 4 CV mouse model of muscular dystrophy by induced exon skipping.

Mitrpant C, Fletcher S, Iversen PL, Wilton SD.

J Gene Med. 2009 Jan;11(1):46-56. doi: 10.1002/jgm.1265.

PMID:
19006096
4.

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide.

Fletcher S, Honeyman K, Fall AM, Harding PL, Johnsen RD, Wilton SD.

J Gene Med. 2006 Feb;8(2):207-16.

PMID:
16285002
5.

Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino.

Wu B, Lu P, Benrashid E, Malik S, Ashar J, Doran TJ, Lu QL.

Gene Ther. 2010 Jan;17(1):132-40. doi: 10.1038/gt.2009.120. Epub 2009 Sep 17.

PMID:
19759562
6.

Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.

Moulton HM, Fletcher S, Neuman BW, McClorey G, Stein DA, Abes S, Wilton SD, Buchmeier MJ, Lebleu B, Iversen PL.

Biochem Soc Trans. 2007 Aug;35(Pt 4):826-8. Review.

PMID:
17635157
7.

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers.

Jearawiriyapaisarn N, Moulton HM, Sazani P, Kole R, Willis MS.

Cardiovasc Res. 2010 Feb 1;85(3):444-53. doi: 10.1093/cvr/cvp335. Epub 2009 Oct 8.

8.

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
9.

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles.

Lu QL, Rabinowitz A, Chen YC, Yokota T, Yin H, Alter J, Jadoon A, Bou-Gharios G, Partridge T.

Proc Natl Acad Sci U S A. 2005 Jan 4;102(1):198-203. Epub 2004 Dec 17.

11.

Cell-penetrating peptides enhance systemic delivery of antisense morpholino oligomers.

Moulton HM.

Methods Mol Biol. 2012;867:407-14. doi: 10.1007/978-1-61779-767-5_26.

PMID:
22454076
12.

In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.

Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ, van Ommen GJ, van Deutekom JC, Aartsma-Rus A.

J Gene Med. 2009 Mar;11(3):257-66. doi: 10.1002/jgm.1288.

PMID:
19140108
13.
14.

Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy.

Moulton HM, Moulton JD.

Biochim Biophys Acta. 2010 Dec;1798(12):2296-303. doi: 10.1016/j.bbamem.2010.02.012. Epub 2010 Feb 17.

15.

Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.

Wu B, Lu P, Cloer C, Shaban M, Grewal S, Milazi S, Shah SN, Moulton HM, Lu QL.

Am J Pathol. 2012 Aug;181(2):392-400. doi: 10.1016/j.ajpath.2012.04.006. Epub 2012 Jun 7.

16.

Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.

Malerba A, Thorogood FC, Dickson G, Graham IR.

Hum Gene Ther. 2009 Sep;20(9):955-65. doi: 10.1089/hum.2008.157.

PMID:
19469709
17.

Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

Gebski BL, Mann CJ, Fletcher S, Wilton SD.

Hum Mol Genet. 2003 Aug 1;12(15):1801-11.

PMID:
12874101
18.

A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.

Yin H, Moulton HM, Betts C, Seow Y, Boutilier J, Iverson PL, Wood MJ.

Hum Mol Genet. 2009 Nov 15;18(22):4405-14. doi: 10.1093/hmg/ddp395. Epub 2009 Aug 18.

PMID:
19692354
19.

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
20.

Nanopolymers improve delivery of exon skipping oligonucleotides and concomitant dystrophin expression in skeletal muscle of mdx mice.

Williams JH, Schray RC, Sirsi SR, Lutz GJ.

BMC Biotechnol. 2008 Apr 2;8:35. doi: 10.1186/1472-6750-8-35.

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