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Results: 1 to 20 of 181

1.

Exon-skipping events in candidates for clinical trials of morpholino.

Nakano S, Ozasa S, Yoshioka K, Fujii I, Mitsui K, Nomura K, Kosuge H, Endo F, Matsukura M, Kimura S.

Pediatr Int. 2011 Aug;53(4):524-9. doi: 10.1111/j.1442-200X.2011.03330.x.

PMID:
21342350
[PubMed - indexed for MEDLINE]
2.

Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers.

Popplewell LJ, Malerba A, Dickson G.

Methods Mol Biol. 2012;867:143-67. doi: 10.1007/978-1-61779-767-5_10.

PMID:
22454060
[PubMed - indexed for MEDLINE]
3.

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.

PMID:
17612397
[PubMed - indexed for MEDLINE]
Free PMC Article
4.

Bioinformatic and functional optimization of antisense phosphorodiamidate morpholino oligomers (PMOs) for therapeutic modulation of RNA splicing in muscle.

Popplewell LJ, Graham IR, Malerba A, Dickson G.

Methods Mol Biol. 2011;709:153-78. doi: 10.1007/978-1-61737-982-6_10.

PMID:
21194027
[PubMed - indexed for MEDLINE]
5.

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
[PubMed - indexed for MEDLINE]
6.

[Exon skipping therapy for Duchenne muscular dystrophy by using antisense Morpholino].

Takeda S.

Rinsho Shinkeigaku. 2009 Nov;49(11):856-8. Review. Japanese.

PMID:
20030230
[PubMed - indexed for MEDLINE]
7.

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
[PubMed - indexed for MEDLINE]
8.

Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells.

van Deutekom JC, Bremmer-Bout M, Janson AA, Ginjaar IB, Baas F, den Dunnen JT, van Ommen GJ.

Hum Mol Genet. 2001 Jul 15;10(15):1547-54.

PMID:
11468272
[PubMed - indexed for MEDLINE]
Free Article
9.

Antisense oligo-mediated multiple exon skipping in a dog model of duchenne muscular dystrophy.

Yokota T, Hoffman E, Takeda S.

Methods Mol Biol. 2011;709:299-312. doi: 10.1007/978-1-61737-982-6_20.

PMID:
21194037
[PubMed - indexed for MEDLINE]
10.

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
[PubMed - indexed for MEDLINE]
11.

Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells.

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

Gene Ther. 2004 Sep;11(18):1391-8.

PMID:
15229633
[PubMed - indexed for MEDLINE]
12.

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
[PubMed - indexed for MEDLINE]
13.

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
[PubMed - indexed for MEDLINE]
14.

Antisense PMO found in dystrophic dog model was effective in cells from exon 7-deleted DMD patient.

Saito T, Nakamura A, Aoki Y, Yokota T, Okada T, Osawa M, Takeda S.

PLoS One. 2010 Aug 18;5(8):e12239. doi: 10.1371/journal.pone.0012239.

PMID:
20805873
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense.

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

Am J Hum Genet. 2004 Jan;74(1):83-92. Epub 2003 Dec 16.

PMID:
14681829
[PubMed - indexed for MEDLINE]
Free PMC Article
16.

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
[PubMed - indexed for MEDLINE]
17.

Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.

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

Hum Mol Genet. 2003 Apr 15;12(8):907-14.

PMID:
12668614
[PubMed - indexed for MEDLINE]
Free Article
18.

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
[PubMed - indexed for MEDLINE]
19.

Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy.

van Vliet L, de Winter CL, van Deutekom JC, van Ommen GJ, Aartsma-Rus A.

BMC Med Genet. 2008 Dec 1;9:105. doi: 10.1186/1471-2350-9-105.

PMID:
19046429
[PubMed - indexed for MEDLINE]
Free PMC Article
20.

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
[PubMed - indexed for MEDLINE]

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