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Items: 1 to 50 of 64

1.

Using a State-of-the-Art Toolbox to Evaluate Molecular and Functional Readouts of Antisense Oligonucleotide-Induced Exon Skipping in mdx Mice.

Datson NA, Bijl S, Janson A, Testerink J, van den Eijnde R, Weij R, Puoliväli J, Lehtimäki K, Bragge T, Ahtoniemi T, van Deutekom JC.

Nucleic Acid Ther. 2020 Feb;30(1):50-65. doi: 10.1089/nat.2019.0824. Epub 2019 Dec 10.

PMID:
31821107
2.

Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide.

Kourkouta E, Weij R, González-Barriga A, Mulder M, Verheul R, Bosgra S, Groenendaal B, Puoliväli J, Toivanen J, van Deutekom JCT, Datson NA.

Mol Ther Nucleic Acids. 2019 Sep 6;17:601-614. doi: 10.1016/j.omtn.2019.07.004. Epub 2019 Jul 19.

3.

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.

4.

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.

5.

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.

6.

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.

7.

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.

8.

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.

9.

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.

10.

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.

11.

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
12.

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
13.

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
14.

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.

15.

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.

16.

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.

17.

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.

18.

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.

19.

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
20.

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
21.

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.

PMID:
19258592
22.

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
23.

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.

24.

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.

25.

Local dystrophin restoration with antisense oligonucleotide PRO051.

van Deutekom JC, Janson AA, Ginjaar IB, Frankhuizen WS, Aartsma-Rus A, Bremmer-Bout M, den Dunnen JT, Koop K, van der Kooi AJ, Goemans NM, de Kimpe SJ, Ekhart PF, Venneker EH, Platenburg GJ, Verschuuren JJ, van Ommen GJ.

N Engl J Med. 2007 Dec 27;357(26):2677-86.

26.

Generation and characterization of transgenic mice with the full-length human DMD gene.

't Hoen PA, de Meijer EJ, Boer JM, Vossen RH, Turk R, Maatman RG, Davies KE, van Ommen GJ, van Deutekom JC, den Dunnen JT.

J Biol Chem. 2008 Feb 29;283(9):5899-907. Epub 2007 Dec 13.

27.

Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle.

Arechavala-Gomeza V, Graham IR, Popplewell LJ, Adams AM, Aartsma-Rus A, Kinali M, Morgan JE, van Deutekom JC, Wilton SD, Dickson G, Muntoni F.

Hum Gene Ther. 2007 Sep;18(9):798-810.

PMID:
17767400
28.

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.

29.

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
30.

Entries in the Leiden Duchenne muscular dystrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule.

Aartsma-Rus A, Van Deutekom JC, Fokkema IF, Van Ommen GJ, Den Dunnen JT.

Muscle Nerve. 2006 Aug;34(2):135-44. Review.

PMID:
16770791
31.

Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons.

Aartsma-Rus A, Kaman WE, Weij R, den Dunnen JT, van Ommen GJ, van Deutekom JC.

Mol Ther. 2006 Sep;14(3):401-7. Epub 2006 Jun 6.

32.

Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy.

't Hoen PA, van der Wees CG, Aartsma-Rus A, Turk R, Goyenvalle A, Danos O, Garcia L, van Ommen GJ, den Dunnen JT, van Deutekom JC.

Pharmacogenomics. 2006 Apr;7(3):281-97.

PMID:
16610940
33.

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
34.

Gene therapy: the 'pro-sense' approach to Duchenne muscular dystrophy.

van Deutekom JC.

Eur J Hum Genet. 2005 May;13(5):518-9. No abstract available.

35.

FRG2, an FSHD candidate gene, is transcriptionally upregulated in differentiating primary myoblast cultures of FSHD patients.

Rijkers T, Deidda G, van Koningsbruggen S, van Geel M, Lemmers RJ, van Deutekom JC, Figlewicz D, Hewitt JE, Padberg GW, Frants RR, van der Maarel SM.

J Med Genet. 2004 Nov;41(11):826-36.

36.

Large-scale gene expression analysis of human skeletal myoblast differentiation.

Sterrenburg E, Turk R, 't Hoen PA, van Deutekom JC, Boer JM, van Ommen GJ, den Dunnen JT.

Neuromuscul Disord. 2004 Sep;14(8-9):507-18.

PMID:
15336692
37.

Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides.

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

Mol Ther. 2004 Aug;10(2):232-40.

38.

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
39.

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.

40.

Advances in Duchenne muscular dystrophy gene therapy.

van Deutekom JC, van Ommen GJ.

Nat Rev Genet. 2003 Oct;4(10):774-83. Review.

PMID:
14526374
41.

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
42.

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
43.

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
44.

Extended tropism of an adenoviral vector does not circumvent the maturation-dependent transducibility of mouse skeletal muscle.

van Deutekom JC, Cao B, Pruchnic R, Wickham TJ, Kovesdi I, Huard J.

J Gene Med. 1999 Nov-Dec;1(6):393-9.

PMID:
10753064
45.

Dystrophin nonsense mutation induces different levels of exon 29 skipping and leads to variable phenotypes within one BMD family.

Ginjaar IB, Kneppers AL, v d Meulen JD, Anderson LV, Bremmer-Bout M, van Deutekom JC, Weegenaar J, den Dunnen JT, Bakker E.

Eur J Hum Genet. 2000 Oct;8(10):793-6.

46.

Identification of a novel beta-tubulin subfamily with one member (TUBB4Q) located near the telomere of chromosome region 4q35.

van Geel M, van Deutekom JC, van Staalduinen A, Lemmers RJ, Dickson MC, Hofker MH, Padberg GW, Hewitt JE, de Jong PJ, Frants RR.

Cytogenet Cell Genet. 2000;88(3-4):316-21.

PMID:
10828619
47.

Development of approaches to improve cell survival in myoblast transfer therapy.

Qu Z, Balkir L, van Deutekom JC, Robbins PD, Pruchnic R, Huard J.

J Cell Biol. 1998 Sep 7;142(5):1257-67.

48.

Inter- and intrachromosomal sub-telomeric rearrangements on 4q35: implications for facioscapulohumeral muscular dystrophy (FSHD) aetiology and diagnosis.

Lemmers RJ, van der Maarel SM, van Deutekom JC, van der Wielen MJ, Deidda G, Dauwerse HG, Hewitt J, Hofker M, Bakker E, Padberg GW, Frants RR.

Hum Mol Genet. 1998 Aug;7(8):1207-14.

PMID:
9668160
49.

Implications of maturation for viral gene delivery to skeletal muscle.

van Deutekom JC, Floyd SS, Booth DK, Oligino T, Krisky D, Marconi P, Glorioso JC, Huard J.

Neuromuscul Disord. 1998 May;8(3-4):135-48.

PMID:
9631393
50.

Muscle maturation: implications for gene therapy.

van Deutekom JC, Hoffman EP, Huard J.

Mol Med Today. 1998 May;4(5):214-20. Review.

PMID:
9612801

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