Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model
Heemskerk, Hans, de Winter, Christa, van Kuik, Petra, Heuvelmans, Niki, Sabatelli, Patrizia, Rimessi, Paola, Braghetta, Paola, van Ommen, Gert-Jan B, de Kimpe, Sjef, Ferlini, Alessandra, Aartsma-Rus, Annemieke, van Deutekom, Judith CT
Published in Molecular therapy (01.06.2010)
Published in Molecular therapy (01.06.2010)
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Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy
Aartsma-Rus, Annemieke, Janson, Anneke A M, van Ommen, Gert-Jan B, van Deutekom, Judith C T
Published in BMC medical genetics (05.07.2007)
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Design and Analysis of Effects of Triplet Repeat Oligonucleotides in Cell Models for Myotonic Dystrophy
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Published in Molecular therapy. Nucleic acids (01.03.2013)
Published in Molecular therapy. Nucleic acids (01.03.2013)
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Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy
t Hoen, Peter AC, van der Wees, Caroline GC, Aartsma-Rus, Annemieke, Turk, Rolf, Goyenvalle, Aurélie, Danos, Olivier, Garcia, Luis, van Ommen, Gert-Jan B, den Dunnen, Johan T, van Deutekom, Judith CT
Published in Pharmacogenomics (01.04.2006)
Published in Pharmacogenomics (01.04.2006)
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Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms
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Published in Molecular therapy (01.03.2009)
Published in Molecular therapy (01.03.2009)
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Targeting several CAG expansion diseases by a single antisense oligonucleotide
Evers, Melvin M, Pepers, Barry A, van Deutekom, Judith C T, Mulders, Susan A M, den Dunnen, Johan T, Aartsma-Rus, Annemieke, van Ommen, Gert-Jan B, van Roon-Mom, Willeke M C
Published in PloS one (01.09.2011)
Published in PloS one (01.09.2011)
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Intracellular Distribution and Nuclear Activity of Antisense Oligonucleotides After Unassisted Uptake in Myoblasts and Differentiated Myotubes In Vitro
González-Barriga, Anchel, Nillessen, Bram, Kranzen, Julia, van Kessel, Ingeborg D.G., Croes, Huib J.E., Aguilera, Begoña, de Visser, Peter C., Datson, Nicole A., Mulders, Susan A.M., van Deutekom, Judith C.T., Wieringa, Bé, Wansink, Derick G.
Published in Nucleic acid therapeutics (01.06.2017)
Published in Nucleic acid therapeutics (01.06.2017)
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Dystrophin nonsense mutation induces different levels of exon 29 skipping and leads to variable phenotypes within one BMD family
Ginjaar, Ieke B, Kneppers, Alexander LJ, v d Meulen, Jan-Douwe M, Anderson, Louise VB, Bremmer-Bout, Mattie, van Deutekom, Judith CT, Weegenaar, Jitske, den Dunnen, Johan T, Bakker, Egbert
Published in European journal of human genetics : EJHG (03.10.2000)
Published in European journal of human genetics : EJHG (03.10.2000)
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Peptide Conjugation of 2′-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in mdx Mice
Jirka, Silvana M.G., Heemskerk, Hans, Tanganyika-de Winter, Christa L., Muilwijk, Daan, Pang, Kar Him, de Visser, Peter C., Janson, Anneke, Karnaoukh, Tatyana G., Vermue, Rick, ‘t Hoen, Peter A.C., van Deutekom, Judith C.T., Aguilera, Begoña, Aartsma-Rus, Annemieke
Published in Nucleic acid therapeutics (01.02.2014)
Published in Nucleic acid therapeutics (01.02.2014)
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Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy
Heemskerk, Hans, de Winter, Christa L., van Ommen, Gert-Jan B., van Deutekom, Judith C.T., Aartsma-Rus, Annemieke
Published in Annals of the New York Academy of Sciences (01.09.2009)
Published in Annals of the New York Academy of Sciences (01.09.2009)
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Dose-Dependent Pharmacokinetic Profiles of 2′-O-Methyl Phosphorothioate Antisense Oligonucleotidesin mdx Mice
Verhaart, Ingrid E.C., Tanganyika-de Winter, Christa L., Karnaoukh, Tatyana G., Kolfschoten, Ingrid G.M., de Kimpe, Sjef J., van Deutekom, Judith C.T., Aartsma-Rus, Annemieke
Published in Nucleic acid therapeutics (01.06.2013)
Published in Nucleic acid therapeutics (01.06.2013)
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Generation and Characterization of Transgenic Mice with the Full-length Human DMD Gene
't Hoen, Peter A.C., de Meijer, Emile J., Boer, Judith M., Vossen, Rolf H.A.M., Turk, Rolf, Maatman, Ronald G.H.J., Davies, Kay E., van Ommen, Gert-Jan B., van Deutekom, Judith C.T., den Dunnen, Johan T.
Published in The Journal of biological chemistry (29.02.2008)
Published in The Journal of biological chemistry (29.02.2008)
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Exon skipping for DMD
Aartsma-Rus, Annemieke, Verschuuren, Jan JGM, Campion, Giles V, van Ommen, Gert-jan B, van Deutekom, Judith CT
Published in Orphanet journal of rare diseases (01.01.2012)
Published in Orphanet journal of rare diseases (01.01.2012)
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Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide
Kourkouta, Eleni, Weij, Rudie, González-Barriga, Anchel, Mulder, Melissa, Verheul, Ruurd, Bosgra, Sieto, Groenendaal, Bas, Puoliväli, Jukka, Toivanen, Jussi, van Deutekom, Judith C.T., Datson, Nicole A.
Published in Molecular therapy. Nucleic acids (06.09.2019)
Published in Molecular therapy. Nucleic acids (06.09.2019)
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L2 The expanded cag repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the R6/2 and Q175 HD mouse brain
González-Barriga, Anchel, Kourkouta, Eleni, Kontkanen, Outi, Heikkinen, Taneli, Lehtimäki, Kimmo, Deutekom, Judith CT van, Datson, Nicole A
Published in Journal of neurology, neurosurgery and psychiatry (01.09.2016)
Published in Journal of neurology, neurosurgery and psychiatry (01.09.2016)
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908. Design and Optimization of U7snRNAs for Skipping of Exon 51 in DMD: Promising Tools for Future Clinical Trials
Goyenvalle, Aurelie, Vulin, Adeline, Lorain, Stephanie, Aartsma-Rus, Annemieke, van Deutekom, Judith C.T., Danos, Olivier, Garcia, Luis
Published in Molecular therapy (01.05.2006)
Published in Molecular therapy (01.05.2006)
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Exonic sequences provide better targets for antisense oligonucleotides than splice site sequences in the modulation of Duchenne muscular dystrophy splicing
Aartsma-Rus, Annemieke, Houlleberghs, Hellen, van Deutekom, Judith C T, van Ommen, Gert-Jan B, 't Hoen, Peter A C
Published in Oligonucleotides (01.04.2010)
Published in Oligonucleotides (01.04.2010)
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Dystrophin nonsense mutation induces different levels of exon29 skipping and leads to variable phenotypes within one BMD family
Ginjaar, Ieke B, Kneppers, Alexander LJ, v d Meulen, Jan-Douwe M, Anderson, Louise VB, Bremmer-Bout, Mattie, van Deutekom, Judith CT, Weegenaar, Jitske, den Dunnen, Johan T, Bakker, Egbert
Published in European journal of human genetics : EJHG (01.10.2000)
Published in European journal of human genetics : EJHG (01.10.2000)
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Journal Article
Dystrophin nonsense mutation induces different levels of exon 29 skipping and leads to variable phenotypes within one BMD family
Ginjaar, I B, Kneppers, A L, v d Meulen, J D, Anderson, L V, Bremmer-Bout, M, van Deutekom, J C, Weegenaar, J, den Dunnen, J T, Bakker, E
Published in European journal of human genetics : EJHG (01.10.2000)
Published in European journal of human genetics : EJHG (01.10.2000)
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