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

by Ruurd C Verheul, Judith C T van Deutekom, Nicole A Datson
Published 2016-01-01

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

by Nicole A Datson, Anchel González-Barriga, Eleni Kourkouta, Rudie Weij, Jeroen van de Giessen, Susan Mulders, Outi Kontkanen, Taneli Heikkinen, Kimmo Lehtimäki, Judith C T van Deutekom
Published 2017-01-01

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

by Eleni Kourkouta, Rudie Weij, Anchel González-Barriga, Melissa Mulder, Ruurd Verheul, Sieto Bosgra, Bas Groenendaal, Jukka Puoliväli, Jussi Toivanen, Judith C.T. van Deutekom, Nicole A. Datson
Published 2019-09-01

Targeting several CAG expansion diseases by a single antisense oligonucleotide.

by Melvin M Evers, Barry A Pepers, Judith C T van Deutekom, Susan A M Mulders, Johan T den Dunnen, Annemieke Aartsma-Rus, Gert-Jan B van Ommen, Willeke M C van Roon-Mom
Published 2011-01-01

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

by Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus
Published 2014-01-01

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

by Anchel González-Barriga, Julia Kranzen, Huib J E Croes, Suzanne Bijl, Walther J A A van den Broek, Ingeborg D G van Kessel, Baziel G M van Engelen, Judith C T van Deutekom, Bé Wieringa, Susan A M Mulders, Derick G Wansink
Published 2015-01-01