The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain von Datson, Nicole A, González-Barriga, Anchel, Kourkouta, Eleni, Weij, Rudie, van de Giessen, Jeroen, Mulders, Susan, Kontkanen, Outi, Heikkinen, Taneli, Lehtimäki, Kimmo, van Deutekom, Judith C T

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Targeting several CAG expansion diseases by a single antisense oligonucleotide von 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

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Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy von Verheul, Ruurd C, van Deutekom, Judith C T, Datson, Nicole A

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Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model von 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

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Advances in Duchenne muscular dystrophy gene therapy von van Deutekom, Judith C. T, van Ommen, Gert-Jan B

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Cell membrane integrity in myotonic dystrophy type 1: implications for therapy von González-Barriga, Anchel, Kranzen, Julia, Croes, Huib J E, Bijl, Suzanne, van den Broek, Walther J A A, van Kessel, Ingeborg D G, van Engelen, Baziel G M, van Deutekom, Judith C T, Wieringa, Bé, Mulders, Susan A M, Wansink, Derick G

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Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy von van Vliet, Laura, de Winter, Christa L, van Deutekom, Judith C T, van Ommen, Gert-Jan B, Aartsma-Rus, Annemieke

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Entries in the Leiden Duchenne muscular dystrophy mutation database: An overview of mutation types and paradoxical cases that confirm the reading-frame rule von Aartsma-Rus, Annemieke, Van Deutekom, Judith C. T., Fokkema, Ivo F., Van Ommen, Gert-Jan B., Den Dunnen, Johan T.

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Generation and Characterization of Transgenic Mice with the Full-length Human DMD Gene von '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.

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

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In vivo comparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping von Heemskerk, Hans A., de Winter, Christa L., de Kimpe, Sjef J., van Kuik-Romeijn, Petra, Heuvelmans, Niki, Platenburg, Gerard J., van Ommen, Gert-Jan B., van Deutekom, Judith C. T., Aartsma-Rus, Annemieke

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Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy von Aartsma-Rus, Annemieke, Janson, Anneke A M, van Ommen, Gert-Jan B, van Deutekom, Judith C T

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Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms von Aartsma-Rus, Annemieke, van Vliet, Laura, Hirschi, Marscha, Janson, Anneke AM, Heemskerk, Hans, de Winter, Christa L, de Kimpe, Sjef, van Deutekom, Judith CT, 't Hoen, Peter AC, van Ommen, Gert-Jan B

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

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Peptide Conjugation of 2′-O-methyl Phosphorothioate Antisense Oligonucleotides Enhances Cardiac Uptake and Exon Skipping in mdx Mice von 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

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Prednisolone Treatment Does Not Interfere with 2-'-O-Methyl Phosphorothioate Antisense-Mediated Exon Skipping in Duchenne Muscular Dystrophy von VERHAART, Ingrid E. C, HEEMSKERK, Hans, KARNAOUKH, Tatyana G, KOLFSCHOTEN, Ingrid G. M, VROON, Anne, OMMEN, Gert-Jan B. Van, DEUTEKOM, Judith C. T. Van, AARTSMA-RUS, Annemieke

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Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy von Heemskerk, Hans, de Winter, Christa L., van Ommen, Gert-Jan B., van Deutekom, Judith C.T., Aartsma-Rus, Annemieke

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The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice von Verhaart, Ingrid E C, van Vliet-van den Dool, Laura, Sipkens, Jessica A, de Kimpe, Sjef J, Kolfschoten, Ingrid G M, van Deutekom, Judith C T, Liefaard, Lia, Ridings, Jim E, Hood, Steve R, Aartsma-Rus, Annemieke

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Dose-Dependent Pharmacokinetic Profiles of 2′-O-Methyl Phosphorothioate Antisense Oligonucleotidesin mdx Mice von 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

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Design and Analysis of Effects of Triplet Repeat Oligonucleotides in Cell Models for Myotonic Dystrophy von González-Barriga, Anchel, Mulders, Susan AM, van de Giessen, Jeroen, Hooijer, Jeroen D, Bijl, Suzanne, van Kessel, Ingeborg DG, van Beers, Josee, van Deutekom, Judith CT, Fransen, Jack AM, Wieringa, Bé, Wansink, Derick G

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