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

od Verheul, Ruurd C., van Deutekom, Judith C. T., Datson, Nicole A.
Wydane w PLoS One (2016)

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

od Beekman, Chantal, Janson, Anneke A., Baghat, Aabed, van Deutekom, Judith C., Datson, Nicole A.
Wydane w PLoS One (2018)

Development of Approaches to Improve Cell Survival in Myoblast Transfer Therapy

od Qu, Zhuqing, Balkir, Levent, van Deutekom, Judith C.T., Robbins, Paul D., Pruchnic, Ryan, Huard, Johnny
Wydane 1998

A Sensitive, Reproducible and Objective Immunofluorescence Analysis Method of Dystrophin in Individual Fibers in Samples from Patients with Duchenne Muscular Dystrophy

od Beekman, Chantal, Sipkens, Jessica A., Testerink, Janwillem, Giannakopoulos, Stavros, Kreuger, Dyonne, van Deutekom, Judith C., Campion, Giles V., de Kimpe, Sjef J., Lourbakos, Afrodite
Wydane 2014

Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense

od Aartsma-Rus, Annemieke, Janson, Anneke A. M., Kaman, Wendy E., Bremmer-Bout, Mattie, van Ommen, Gert-Jan B., den Dunnen, Johan T., van Deutekom, Judith C. T.
Wydane 2004

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

od 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.
Wydane w PLoS One (2017)

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

od 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.
Wydane w Mol Ther Nucleic Acids (2019)

Prednisolone Treatment Does Not Interfere with 2′-O-Methyl Phosphorothioate Antisense-Mediated Exon Skipping in Duchenne Muscular Dystrophy

od Verhaart, Ingrid E.C., Heemskerk, Hans, Karnaoukh, Tatyana G., Kolfschoten, Ingrid G.M., Vroon, Anne, van Ommen, Gert-Jan B., van Deutekom, Judith C.T., Aartsma-Rus, Annemieke
Wydane 2012

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

od Datson, Nicole A., Bijl, Suzanne, Janson, Anneke, Testerink, Janwillem, van den Eijnde, Rani, Weij, Rudie, Puoliväli, Jukka, Lehtimäki, Kimmo, Bragge, Timo, Ahtoniemi, Toni, van Deutekom, Judith C.
Wydane w Nucleic Acid Ther (2020)

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

od Goemans, Nathalie M., Tulinius, Már, van den Hauwe, Marleen, Kroksmark, Anna-Karin, Buyse, Gunnar, Wilson, Rosamund J., van Deutekom, Judith C., de Kimpe, Sjef J., Lourbakos, Afrodite, Campion, Giles
Wydane w PLoS One (2016)

Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide

od 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.
Wydane 2011

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

od 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
Wydane 2014

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

od 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.
Wydane w Nucleic Acid Ther (2017)

Cell Membrane Integrity in Myotonic Dystrophy Type 1: Implications for Therapy

od 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.
Wydane w PLoS One (2015)