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Items: 1 to 20 of 185

1.

Multiple exon skipping strategies to by-pass dystrophin mutations.

Adkin CF, Meloni PL, Fletcher S, Adams AM, Muntoni F, Wong B, Wilton SD.

Neuromuscul Disord. 2012 Apr;22(4):297-305. doi: 10.1016/j.nmd.2011.10.007. Epub 2011 Dec 17.

2.

Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations.

Forrest S, Meloni PL, Muntoni F, Kim J, Fletcher S, Wilton SD.

Neuromuscul Disord. 2010 Dec;20(12):810-6. doi: 10.1016/j.nmd.2010.07.276.

3.

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

Aartsma-Rus A, Fokkema I, Verschuuren J, Ginjaar I, van Deutekom J, van Ommen GJ, den Dunnen JT.

Hum Mutat. 2009 Mar;30(3):293-9. doi: 10.1002/humu.20918. Review.

PMID:
19156838
4.

Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching.

Fragall CT, Adams AM, Johnsen RD, Kole R, Fletcher S, Wilton SD.

BMC Med Genet. 2011 Oct 20;12:141. doi: 10.1186/1471-2350-12-141.

5.

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy.

Aartsma-Rus A, Janson AA, van Ommen GJ, van Deutekom JC.

BMC Med Genet. 2007 Jul 5;8:43.

6.

Characterization of a complex Duchenne muscular dystrophy-causing dystrophin gene inversion and restoration of the reading frame by induced exon skipping.

Madden HR, Fletcher S, Davis MR, Wilton SD.

Hum Mutat. 2009 Jan;30(1):22-8. doi: 10.1002/humu.20806.

PMID:
18570328
7.

Antisense oligonucleotides, exon skipping and the dystrophin gene transcript.

Wilton SD, Fletcher S.

Acta Myol. 2005 Dec;24(3):222-9. Review.

PMID:
16629057
8.

[Mutation-specific treatments for Duchenne muscular dystrophy].

Matsuo M, Takeshima Y.

Brain Nerve. 2009 Aug;61(8):915-22. Review. Japanese.

PMID:
19697880
9.

Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

Aartsma-Rus A, Bremmer-Bout M, Janson AA, den Dunnen JT, van Ommen GJ, van Deutekom JC.

Neuromuscul Disord. 2002 Oct;12 Suppl 1:S71-7.

PMID:
12206800
10.

Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.

Anthony K, Arechavala-Gomeza V, Ricotti V, Torelli S, Feng L, Janghra N, Tasca G, Guglieri M, Barresi R, Armaroli A, Ferlini A, Bushby K, Straub V, Ricci E, Sewry C, Morgan J, Muntoni F.

JAMA Neurol. 2014 Jan;71(1):32-40. doi: 10.1001/jamaneurol.2013.4908.

PMID:
24217213
11.

Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides.

Echigoya Y, Yokota T.

Nucleic Acid Ther. 2014 Feb;24(1):57-68. doi: 10.1089/nat.2013.0451. Epub 2013 Dec 31. Review.

PMID:
24380394
12.

Exon skipping-mediated dystrophin reading frame restoration for small mutations.

Spitali P, Rimessi P, Fabris M, Perrone D, Falzarano S, Bovolenta M, Trabanelli C, Mari L, Bassi E, Tuffery S, Gualandi F, Maraldi NM, Sabatelli-Giraud P, Medici A, Merlini L, Ferlini A.

Hum Mutat. 2009 Nov;30(11):1527-34. doi: 10.1002/humu.21092.

PMID:
19760747
13.

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
14.

Deletion of Dystrophin In-Frame Exon 5 Leads to a Severe Phenotype: Guidance for Exon Skipping Strategies.

Toh ZY, Thandar Aung-Htut M, Pinniger G, Adams AM, Krishnaswarmy S, Wong BL, Fletcher S, Wilton SD.

PLoS One. 2016 Jan 8;11(1):e0145620. doi: 10.1371/journal.pone.0145620. eCollection 2016.

15.

Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse.

Fletcher S, Honeyman K, Fall AM, Harding PL, Johnsen RD, Steinhaus JP, Moulton HM, Iversen PL, Wilton SD.

Mol Ther. 2007 Sep;15(9):1587-92. Epub 2007 Jun 19.

16.

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
17.

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy.

Béroud C, Tuffery-Giraud S, Matsuo M, Hamroun D, Humbertclaude V, Monnier N, Moizard MP, Voelckel MA, Calemard LM, Boisseau P, Blayau M, Philippe C, Cossée M, Pagès M, Rivier F, Danos O, Garcia L, Claustres M.

Hum Mutat. 2007 Feb;28(2):196-202.

PMID:
17041910
18.

Exon skipping and Duchenne muscular dystrophy: hope, hype and how feasible?

Wilton SD, Fletcher S.

Neurol India. 2008 Jul-Sep;56(3):254-62. Review.

19.

Overview on DMD exon skipping.

Aartsma-Rus A.

Methods Mol Biol. 2012;867:97-116. doi: 10.1007/978-1-61779-767-5_7. Review.

PMID:
22454057
20.

Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?

Yokota T, Duddy W, Echigoya Y, Kolski H.

Expert Opin Biol Ther. 2012 Sep;12(9):1141-52. doi: 10.1517/14712598.2012.693469. Epub 2012 Jun 1. Review.

PMID:
22650324

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