Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23 by Annemieke Aartsma-Rus

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Multiomic characterization of disease progression in mice lacking dystrophin. by Mirko Signorelli, Roula Tsonaka, Annemieke Aartsma-Rus, Pietro Spitali

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Measuring DNA hybridization using fluorescent DNA-stabilized silver clusters to investigate mismatch effects on therapeutic oligonucleotides by Donny de Bruin, Nelli Bossert, Annemieke Aartsma-Rus, Dirk Bouwmeester

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DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon by Remko Goossens, Nisha Verwey, Yavuz Ariyurek, Fred Schnell, Annemieke Aartsma-Rus

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At the heart of genetic disease: an interview with Elizabeth McNally by Elizabeth McNally, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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To dystrophin and beyond: an interview with Louis Kunkel by Louis M. Kunkel, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders by Marlen C. Lauffer, Willeke van Roon-Mom, Annemieke Aartsma-Rus, N = 1 Collaborative

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Identification of suitable qPCR reference genes for the normalization of gene expression in the BL10-mdx and D2-mdx mouse models of Duchenne muscular dystrophy. by Kayleigh Putker, Anne-Fleur Schneider, Davy Van De Vijver, John Hildyard, Annemieke Aartsma-Rus, Maaike van Putten

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Histone deacetylase inhibitors improve antisense-mediated exon-skipping efficacy in mdx mice by Flavien Bizot, Remko Goossens, Thomas Tensorer, Sergei Dmitriev, Luis Garcia, Annemieke Aartsma-Rus, Pietro Spitali, Aurélie Goyenvalle

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Interactions among ryanodine receptor isotypes contribute to muscle fiber type development and function by Alexis A. Chagovetz, Dana Klatt Shaw, Erin Ritchie, Kazuyuki Hoshijima, David J. Grunwald, Annemieke Aartsma-Rus, James Dowling, Maaike van Putten

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P171: Developing an approach to screening rare genetic diagnoses for amenability to bespoke genetic therapy development by David Cheerie, Marlen Lauffer, Danique Beijer, Matthis Synofzik, Annemieke Aartsma-Rus, Michael Szego, Kimberly Amburgey, Brian Kalish, James Dowling, Gregory Costain

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A modified diet does not ameliorate muscle pathology in a mouse model for Duchenne muscular dystrophy. by Ingrid E C Verhaart, Davy van de Vijver, Joke W Boertje-van der Meulen, Kayleigh Putker, Kevin Adamzek, Annemieke Aartsma-Rus, Maaike van Putten

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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

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Networking to Optimize <i>Dmd</i> exon 53 Skipping in the Brain of <i>mdx52</i> Mouse Model by Mathilde Doisy, Ophélie Vacca, Claire Fergus, Talia Gileadi, Minou Verhaeg, Amel Saoudi, Thomas Tensorer, Luis Garcia, Vincent P. Kelly, Federica Montanaro, Jennifer E. Morgan, Maaike van Putten, Annemieke Aartsma-Rus, Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle

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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

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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

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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

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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

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Delivery is key: lessons learnt from developing splice‐switching antisense therapies by Caroline Godfrey, Lourdes R Desviat, Bård Smedsrød, France Piétri‐Rouxel, Michela A Denti, Petra Disterer, Stéphanie Lorain, Gisela Nogales‐Gadea, Valentina Sardone, Rayan Anwar, Samir EL Andaloussi, Taavi Lehto, Bernard Khoo, Camilla Brolin, Willeke MC van Roon‐Mom, Aurélie Goyenvalle, Annemieke Aartsma‐Rus, Virginia Arechavala‐Gomeza

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Diffusion‐tensor magnetic resonance imaging captures increased skeletal muscle fibre diameters in Becker muscular dystrophy by Donnie Cameron, Tooba Abbassi‐Daloii, Laura G.M. Heezen, Nienke M. van deVelde, Zaïda Koeks, Thom T.J. Veeger, Melissa T. Hooijmans, Salma elAbdellaoui, Sjoerd G. vanDuinen, Jan J.G.M. Verschuuren, Maaike vanPutten, Annemieke Aartsma‐Rus, Vered Raz, Pietro Spitali, Erik H. Niks, Hermien E. Kan

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