Antisense-oligonucleotide mediated exon skipping in activin-receptor-like kinase 2: inhibiting the receptor that is overactive in fibrodysplasia ossificans progressiva.

by Songting Shi, Jie Cai, David J J de Gorter, Gonzalo Sanchez-Duffhues, Dwi U Kemaladewi, Willem M H Hoogaars, Annemieke Aartsma-Rus, Peter A C 't Hoen, Peter ten Dijke
Published 2013-01-01

Longitudinal serum biomarker screening identifies malate dehydrogenase 2 as candidate prognostic biomarker for Duchenne muscular dystrophy

by Mirko Signorelli, Burcu Ayoglu, Camilla Johansson, Hanns Lochmüller, Volker Straub, Francesco Muntoni, Erik Niks, Roula Tsonaka, Anja Persson, Annemieke Aartsma‐Rus, Peter Nilsson, Cristina Al‐Khalili Szigyarto, Pietro Spitali
Published 2020-04-01

Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

by Silvana M G Jirka, Christa L Tanganyika-de Winter, Joke W Boertje-van der Meulen, Maaike van Putten, Monika Hiller, Rick Vermue, Peter C de Visser, Annemieke Aartsma-Rus
Published 2015-01-01

Targeting TGF-β Signaling by Antisense Oligonucleotide-mediated Knockdown of TGF-β Type I Receptor

by Dwi U Kemaladewi, Svitlana Pasteuning, Joke W van der Meulen, Sandra H van Heiningen, Gert-Jan van Ommen, Peter ten Dijke, Annemieke Aartsma-Rus, Peter AC 't Hoen, Willem M Hoogaars
Published 2014-01-01

A dystrophic Duchenne mouse model for testing human antisense oligonucleotides.

by Marcel Veltrop, Laura van Vliet, Margriet Hulsker, Jill Claassens, Conny Brouwers, Cor Breukel, Jos van der Kaa, Margot M Linssen, Johan T den Dunnen, Sjef Verbeek, Annemieke Aartsma-Rus, Maaike van Putten
Published 2018-01-01

A comparison of the bone and growth phenotype of mdx, mdx:Cmah−/− and mdx:Utrn+/− murine models with the C57BL/10 wild-type mouse

by Claire L. Wood, Karla J. Suchacki, Rob van ’t Hof, Will P. Cawthorn, Scott Dillon, Volker Straub, Sze Choong Wong, Syed F. Ahmed, Colin Farquharson, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten
Published 2020-02-01

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

Inhibition of IL-1 Signaling by Antisense Oligonucleotide-mediated Exon Skipping of IL-1 Receptor Accessory Protein (IL-1RAcP)

by A Seda Yılmaz-Eliş, Annemieke Aartsma-Rus, Peter AC 't Hoen, Huma Safdar, Cor Breukel, Bart JM van Vlijmen, Judith van Deutekom, Sjef de Kimpe, Gert-Jan van Ommen, J Sjef Verbeek
Published 2013-01-01

Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon

by Melvin M. Evers, Hoang-Dai Tran, Ioannis Zalachoras, Barry A. Pepers, Onno C. Meijer, Johan T. den Dunnen, Gert-Jan B. van Ommen, Annemieke Aartsma-Rus, Willeke M.C. van Roon-Mom
Published 2013-10-01

Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model.

by Alper Yavas, Rudie Weij, Maaike van Putten, Eleni Kourkouta, Chantal Beekman, Jukka Puoliväli, Timo Bragge, Toni Ahtoniemi, Jeroen Knijnenburg, Marlies Elisabeth Hoogenboom, Yavuz Ariyurek, Annemieke Aartsma-Rus, Judith van Deutekom, Nicole Datson
Published 2020-01-01

The effects of low levels of dystrophin on mouse muscle function and pathology.

by Maaike van Putten, Margriet Hulsker, Vishna Devi Nadarajah, Sandra H van Heiningen, Ella van Huizen, Maarten van Iterson, Peter Admiraal, Tobias Messemaker, Johan T den Dunnen, Peter A C 't Hoen, Annemieke Aartsma-Rus
Published 2012-01-01

Integrated lipidomic and transcriptomic analyses identify altered nerve triglycerides in mouse models of prediabetes and type 2 diabetes

by Phillipe D. O'Brien, Kai Guo, Stephanie A. Eid, Amy E. Rumora, Lucy M. Hinder, John M. Hayes, Faye E. Mendelson, Junguk Hur, Eva L. Feldman, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten
Published 2020-02-01

BMP antagonists enhance myogenic differentiation and ameliorate the dystrophic phenotype in a DMD mouse model

by SongTing Shi, Willem M.H. Hoogaars, David J.J. de Gorter, Sandra H. van Heiningen, Herbert Y. Lin, Charles C. Hong, Dwi U. Kemaladewi, Annemieke Aartsma-Rus, Peter ten Dijke, Peter A.C. 't Hoen
Published 2011-02-01

Modelling the pathogenesis of X-linked distal hereditary motor neuropathy using patient-derived iPSCs

by Gonzalo Perez-Siles, Anthony Cutrupi, Melina Ellis, Jakob Kuriakose, Sharon La Fontaine, Di Mao, Motonari Uesugi, Reinaldo I. Takata, Carlos E. Speck-Martins, Garth Nicholson, Marina L. Kennerson, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten
Published 2020-02-01

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

Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology

by Tirsa L.E. vanWestering, Yulia Lomonosova, Anna M.L. Coenen‐Stass, Corinne A. Betts, Amarjit Bhomra, Margriet Hulsker, Lucy E. Clark, Graham McClorey, Annemieke Aartsma‐Rus, Maaike vanPutten, Matthew J.A. Wood, Thomas C. Roberts
Published 2020-04-01

Peripheral blood transcriptome profiling enables monitoring disease progression in dystrophic mice and patients

by Mirko Signorelli, Mitra Ebrahimpoor, Olga Veth, Kristina Hettne, Nisha Verwey, Raquel García‐Rodríguez, Christa L Tanganyika‐deWinter, Luz B Lopez Hernandez, Rosa Escobar Cedillo, Benjamín Gómez Díaz, Olafur T Magnusson, Hailiang Mei, Roula Tsonaka, Annemieke Aartsma‐Rus, Pietro Spitali
Published 2021-04-01

Novel Ex Vivo Culture Method for the Study of Dupuytren's Disease: Effects of TGFβ Type 1 Receptor Modulation by Antisense Oligonucleotides

by Sofia Karkampouna, Boudewijn PT Kruithof, Peter Kloen, Miryam C Obdeijn, Annelies MA van der Laan, Hans J Tanke, Dwi U Kemaladewi, Willem MH Hoogaars, Peter AC 't Hoen, Annemieke Aartsma-Rus, Ian M Clark, Peter ten Dijke, Marie-José Goumans, Marianna Kruithof-de Julio
Published 2014-01-01

A multicenter comparison of quantification methods for antisense oligonucleotide-induced DMD exon 51 skipping in Duchenne muscular dystrophy cell cultures.

by Monika Hiller, Maria Sofia Falzarano, Iker Garcia-Jimenez, Valentina Sardone, Ruurd C Verheul, Linda Popplewell, Karen Anthony, Estibaliz Ruiz-Del-Yerro, Hana Osman, Jelle J Goeman, Kamel Mamchaoui, George Dickson, Alessandra Ferlini, Francesco Muntoni, Annemieke Aartsma-Rus, Virginia Arechavala-Gomeza, Nicole A Datson, Pietro Spitali
Published 2018-01-01

Tracking disease progression non‐invasively in Duchenne and Becker muscular dystrophies

by Pietro Spitali, Kristina Hettne, Roula Tsonaka, Mohammed Charrout, Janneke van denBergen, Zaïda Koeks, Hermien E. Kan, Melissa T. Hooijmans, Andreas Roos, Volker Straub, Francesco Muntoni, Cristina Al‐Khalili‐Szigyarto, Marleen J.A. Koel‐Simmelink, Charlotte E. Teunissen, Hanns Lochmüller, Erik H. Niks, Annemieke Aartsma‐Rus
Published 2018-08-01