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Items: 18

1.

Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results.

van Putten M, Tanganyika-de Winter C, Bosgra S, Aartsma-Rus A.

Nucleic Acid Ther. 2019 Apr;29(2):92-103. doi: 10.1089/nat.2018.0759. Epub 2019 Jan 23.

2.

Voluntary exercise improves muscle function and does not exacerbate muscle and heart pathology in aged Duchenne muscular dystrophy mice.

Kogelman B, Putker K, Hulsker M, Tanganyika-de Winter C, van der Weerd L, Aartsma-Rus A, van Putten M.

J Mol Cell Cardiol. 2018 Dec;125:29-38. doi: 10.1016/j.yjmcc.2018.10.008. Epub 2018 Oct 16.

3.

Natural disease history of the dy2J mouse model of laminin α2 (merosin)-deficient congenital muscular dystrophy.

Pasteuning-Vuhman S, Putker K, Tanganyika-de Winter CL, Boertje-van der Meulen JW, van Vliet L, Overzier M, Plomp JJ, Aartsma-Rus A, van Putten M.

PLoS One. 2018 May 15;13(5):e0197388. doi: 10.1371/journal.pone.0197388. eCollection 2018.

4.

Cyclic Peptides to Improve Delivery and Exon Skipping of Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy.

Jirka SMG, 't Hoen PAC, Diaz Parillas V, Tanganyika-de Winter CL, Verheul RC, Aguilera B, de Visser PC, Aartsma-Rus AM.

Mol Ther. 2018 Jan 3;26(1):132-147. doi: 10.1016/j.ymthe.2017.10.004. Epub 2017 Oct 12.

5.

Natural disease history of mouse models for limb girdle muscular dystrophy types 2D and 2F.

Pasteuning-Vuhman S, Putker K, Tanganyika-de Winter CL, Boertje-van der Meulen JW, van Vliet L, Overzier M, Plomp JJ, Aartsma-Rus A, van Putten M.

PLoS One. 2017 Aug 10;12(8):e0182704. doi: 10.1371/journal.pone.0182704. eCollection 2017.

6.

Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy.

Jirka SM, Tanganyika-de Winter CL, Boertje-van der Meulen JW, van Putten M, Hiller M, Vermue R, de Visser PC, Aartsma-Rus A.

Mol Ther Nucleic Acids. 2015 Dec 1;4:e265. doi: 10.1038/mtna.2015.39.

7.

Peptide conjugation of 2'-O-methyl phosphorothioate antisense oligonucleotides enhances cardiac uptake and exon skipping in mdx mice.

Jirka SM, Heemskerk H, Tanganyika-de Winter CL, Muilwijk D, Pang KH, de Visser PC, Janson A, Karnaoukh TG, Vermue R, 't Hoen PA, van Deutekom JC, Aguilera B, Aartsma-Rus A.

Nucleic Acid Ther. 2014 Feb;24(1):25-36. doi: 10.1089/nat.2013.0448. Epub 2013 Dec 9.

PMID:
24320790
8.

Dose-dependent pharmacokinetic profiles of 2'-O-methyl phosphorothioate antisense oligonucleotidesin mdx mice.

Verhaart IE, Tanganyika-de Winter CL, Karnaoukh TG, Kolfschoten IG, de Kimpe SJ, van Deutekom JC, Aartsma-Rus A.

Nucleic Acid Ther. 2013 Jun;23(3):228-37. doi: 10.1089/nat.2012.0398. Epub 2013 May 2.

PMID:
23634945
9.

Long-term Exon Skipping Studies With 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in Dystrophic Mouse Models.

Tanganyika-de Winter CL, Heemskerk H, Karnaoukh TG, van Putten M, de Kimpe SJ, van Deutekom J, Aartsma-Rus A.

Mol Ther Nucleic Acids. 2012 Sep 4;1:e44. doi: 10.1038/mtna.2012.38.

10.

Preclinical PK and PD studies on 2'-O-methyl-phosphorothioate RNA antisense oligonucleotides in the mdx mouse model.

Heemskerk H, de Winter C, van Kuik P, Heuvelmans N, Sabatelli P, Rimessi P, Braghetta P, van Ommen GJ, de Kimpe S, Ferlini A, Aartsma-Rus A, van Deutekom JC.

Mol Ther. 2010 Jun;18(6):1210-7. doi: 10.1038/mt.2010.72. Epub 2010 Apr 20.

11.

A 3 months mild functional test regime does not affect disease parameters in young mdx mice.

van Putten M, de Winter C, van Roon-Mom W, van Ommen GJ, 't Hoen PA, Aartsma-Rus A.

Neuromuscul Disord. 2010 Apr;20(4):273-80. doi: 10.1016/j.nmd.2010.02.004. Epub 2010 Mar 21.

PMID:
20307983
12.

Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials.

Popplewell LJ, Adkin C, Arechavala-Gomeza V, Aartsma-Rus A, de Winter CL, Wilton SD, Morgan JE, Muntoni F, Graham IR, Dickson G.

Neuromuscul Disord. 2010 Feb;20(2):102-10. doi: 10.1016/j.nmd.2009.10.013. Epub 2010 Jan 15.

PMID:
20079639
13.

Development of antisense-mediated exon skipping as a treatment for duchenne muscular dystrophy.

Heemskerk H, de Winter CL, van Ommen GJ, van Deutekom JC, Aartsma-Rus A.

Ann N Y Acad Sci. 2009 Sep;1175:71-9. doi: 10.1111/j.1749-6632.2009.04973.x.

PMID:
19796079
14.

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
15.

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.

16.

Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms.

Aartsma-Rus A, van Vliet L, Hirschi M, Janson AA, Heemskerk H, de Winter CL, de Kimpe S, van Deutekom JC, 't Hoen PA, van Ommen GJ.

Mol Ther. 2009 Mar;17(3):548-53. doi: 10.1038/mt.2008.205. Epub 2008 Sep 23.

17.

Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides.

Aartsma-Rus A, Janson AA, Heemskerk JA, De Winter CL, Van Ommen GJ, Van Deutekom JC.

Ann N Y Acad Sci. 2006 Oct;1082:74-6.

PMID:
17145928
18.

Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites.

Aartsma-Rus A, De Winter CL, Janson AA, Kaman WE, Van Ommen GJ, Den Dunnen JT, Van Deutekom JC.

Oligonucleotides. 2005 Dec;15(4):284-97.

PMID:
16396622

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