Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 1 to 20 of 62

1.

Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy.

Beekman C, Janson AA, Baghat A, van Deutekom JC, Datson NA.

PLoS One. 2018 Apr 11;13(4):e0195850. doi: 10.1371/journal.pone.0195850. eCollection 2018.

2.

Intracellular Distribution and Nuclear Activity of Antisense Oligonucleotides After Unassisted Uptake in Myoblasts and Differentiated Myotubes In Vitro.

González-Barriga A, Nillessen B, Kranzen J, van Kessel IDG, Croes HJE, Aguilera B, de Visser PC, Datson NA, Mulders SAM, van Deutekom JCT, Wieringa B, Wansink DG.

Nucleic Acid Ther. 2017 Jun;27(3):144-158. doi: 10.1089/nat.2016.0641. Epub 2017 Apr 4.

3.

The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain.

Datson NA, González-Barriga A, Kourkouta E, Weij R, van de Giessen J, Mulders S, Kontkanen O, Heikkinen T, Lehtimäki K, van Deutekom JC.

PLoS One. 2017 Feb 9;12(2):e0171127. doi: 10.1371/journal.pone.0171127. eCollection 2017.

4.

Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy.

Verheul RC, van Deutekom JC, Datson NA.

PLoS One. 2016 Sep 9;11(9):e0162467. doi: 10.1371/journal.pone.0162467. eCollection 2016.

5.

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study.

Goemans NM, Tulinius M, van den Hauwe M, Kroksmark AK, Buyse G, Wilson RJ, van Deutekom JC, de Kimpe SJ, Lourbakos A, Campion G.

PLoS One. 2016 Sep 2;11(9):e0161955. doi: 10.1371/journal.pone.0161955. eCollection 2016.

6.

Cell membrane integrity in myotonic dystrophy type 1: implications for therapy.

González-Barriga A, Kranzen J, Croes HJ, Bijl S, van den Broek WJ, van Kessel ID, van Engelen BG, van Deutekom JC, Wieringa B, Mulders SA, Wansink DG.

PLoS One. 2015 Mar 23;10(3):e0121556. doi: 10.1371/journal.pone.0121556. eCollection 2015.

7.

A sensitive, reproducible and objective immunofluorescence analysis method of dystrophin in individual fibers in samples from patients with duchenne muscular dystrophy.

Beekman C, Sipkens JA, Testerink J, Giannakopoulos S, Kreuger D, van Deutekom JC, Campion GV, de Kimpe SJ, Lourbakos A.

PLoS One. 2014 Sep 22;9(9):e107494. doi: 10.1371/journal.pone.0107494. eCollection 2014.

8.

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice.

Verhaart IE, van Vliet-van den Dool L, Sipkens JA, de Kimpe SJ, Kolfschoten IG, van Deutekom JC, Liefaard L, Ridings JE, Hood SR, Aartsma-Rus A.

Mol Ther Nucleic Acids. 2014 Feb 18;3:e148. doi: 10.1038/mtna.2014.1.

9.

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
10.

Dystrophin levels and clinical severity in Becker muscular dystrophy patients.

van den Bergen JC, Wokke BH, Janson AA, van Duinen SG, Hulsker MA, Ginjaar HB, van Deutekom JC, Aartsma-Rus A, Kan HE, Verschuuren JJ.

J Neurol Neurosurg Psychiatry. 2014 Jul;85(7):747-53. doi: 10.1136/jnnp-2013-306350. Epub 2013 Nov 29.

PMID:
24292997
11.

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
12.

Design and analysis of effects of triplet repeat oligonucleotides in cell models for myotonic dystrophy.

González-Barriga A, Mulders SA, van de Giessen J, Hooijer JD, Bijl S, van Kessel ID, van Beers J, van Deutekom JC, Fransen JA, Wieringa B, Wansink DG.

Mol Ther Nucleic Acids. 2013 Mar 19;2:e81. doi: 10.1038/mtna.2013.9.

13.

Prednisolone treatment does not interfere with 2'-O-methyl phosphorothioate antisense-mediated exon skipping in Duchenne muscular dystrophy.

Verhaart IE, Heemskerk H, Karnaoukh TG, Kolfschoten IG, Vroon A, van Ommen GJ, van Deutekom JC, Aartsma-Rus A.

Hum Gene Ther. 2012 Mar;23(3):262-73. doi: 10.1089/hum.2011.127. Epub 2012 Jan 26.

14.

Targeting several CAG expansion diseases by a single antisense oligonucleotide.

Evers MM, Pepers BA, van Deutekom JC, Mulders SA, den Dunnen JT, Aartsma-Rus A, van Ommen GJ, van Roon-Mom WM.

PLoS One. 2011;6(9):e24308. doi: 10.1371/journal.pone.0024308. Epub 2011 Sep 1.

15.

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC.

N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23. Erratum in: N Engl J Med. 2011 Oct 6;365(14):1361.

16.

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.

17.

Exonic sequences provide better targets for antisense oligonucleotides than splice site sequences in the modulation of Duchenne muscular dystrophy splicing.

Aartsma-Rus A, Houlleberghs H, van Deutekom JC, van Ommen GJ, 't Hoen PA.

Oligonucleotides. 2010 Apr;20(2):69-77. doi: 10.1089/oli.2009.0215.

PMID:
20377429
18.

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
19.

Antisense-mediated exon skipping to correct IL-12Rbeta1 deficiency in T cells.

van de Vosse E, Verhard EM, de Paus RA, Platenburg GJ, van Deutekom JC, Aartsma-Rus A, van Dissel JT.

Blood. 2009 May 7;113(19):4548-55. doi: 10.1182/blood-2008-12-196220. Epub 2009 Mar 3. Erratum in: Blood. 2009 Nov 12;114(20):4607.

20.

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

Supplemental Content

Loading ...
Support Center