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DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon2023 RNA Biology article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Remko Goossens, Nisha E. Verwey, Yavuz Ariyürek, Fred Schnell, Annemieke M Aartsma-Rus
2citations -
DMD antisense oligonucleotide mediated exon skipping efficiency is affected by flanking intron retention time and target position within the exon2022 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Remko Goossens, Nisha E. Verwey, Yavuz Ariyürek, Fred Schnell, Annemieke M Aartsma-Rus
0citations -
287P3D tissue engineered skeletal muscle to model dystrophinopathies and determine the efficacy of exon skip therapies2025 Neuromuscular Disorders article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders
Liselotte Timp, Marnix Franken, Manuel A.F.V. Gonçalves, Aurélie Goyenvalle, Anne Bigot, Vincent Mouly, +6 more
0citations -
Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model2023 Biomedicines article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Mathilde Doisy, Ophélie Vacca, Claire Fergus, Talia E. Gileadi, Minou A. T. Verhaeg, Amel Saoudi, +10 more
11citations -
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy2023 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 1 patent
J.C.T. van Deutekom, Chantal Beekman, Suzanne Bijl, Sieto Bosgra, Rani van den Eijnde, Dennis Franken, +17 more
19citations -
Newborn screening of duchenne muscular dystrophy specifically targeting deletions amenable to exon-skipping therapy2021 Scientific Reports article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 1 patent
Pablo Beckers, Jean‐Hubert Caberg, Vinciane Dideberg, Tamara Dangouloff, Johan T. den Dunnen, Vincent Bours, +2 more
19citations -
Evaluating exon skipping in the central nervous system in Duchenne muscular dystrophy using spatial transcriptomics2026 iScience article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Sharon I. de Vries, L. Heezen, Ophélie Vacca, Mathilde Doisy, Annemieke M Aartsma-Rus, Maaike van Putten, +3 more
0citations -
Expanding the options for therapeutic exon skipping as a future treatment for USH2A -associated disease by 3D structural modeling of newly formed hybrid domains2026 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders
Lucija Malinar, Sanne Broekman, Daniel T. Rademaker, Anh Q Le, Theo A. Peters, Erik de Vrieze, +3 more
1citations -
Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy2025 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Sofia Stenler, Junyu Huang, Tirsa L.E. van Westering, Anna M. L. Coenen-Stass, Kaarel Krjutškov, Samir EL Andaloussi, +2 more
0citations -
The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience2023 Journal of Neuromuscular Diseases article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Annemieke M Aartsma-Rus, Liesbeth De Waele, Saskia Houwen-Opstal, Janbernd Kirschner, Yvonne D. Krom, Eugenio Maria Mercuri, +4 more
15citations -
Cholesterol Conjugation Strategies to Enhance In Vivo Antisense Oligonucleotide-Mediated Exon Skipping in the Mouse Retina2026 Journal of Ocular Pharmacology and Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery
Peter C. de Visser, Fabian N. Bangel, Dionne Blangé, Herma J.A. Anthonijsz, Monica T. Tartjiono, Wouter Beumer, +1 more
0citations -
Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy2023 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 1 patent
Sarah Engelbeen, Daniel O’Reilly, Ingrid E.C. Verhaart, Maaike van Putten, Vignesh Narayan Hariharan, Matthew R. Hassler, +3 more
16citations -
Evaluating exon-skipping therapies targeting the central nervous system in Duchenne muscular dystrophy using high-resolution spatial transcriptomics2025 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Sharon I. de Vries, L. Heezen, Ophélie Vacca, Mathilde Doisy, Annemieke M Aartsma-Rus, Maaike van Putten, +3 more
0citations -
MOMENT registry: Patients with advanced non-small-cell lung cancer harboring MET exon 14 skipping treated with systemic therapy2025 Journal of Comparative Effectiveness Research article Medicine Lung Cancer Treatments and Mutations Open access
Michael Thomas, Petros Christopoulos, Wade Thomas Iams, Julien Mazières, Alexis Benjamin Cortot, Nir Peled, +8 more
2citations -
Antisense-mediated exon skipping therapy improves neuromuscular junction deficits in a Duchenne muscular dystrophy mouse model2025 Neuromuscular Disorders article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Elizabeth M. van der Pijl, Svetlana Pasteuning-Vuhman, J. W. Boertje-van der Meulen, Erik Harmen Niks, Jan J.G.M. Verschuuren, Annemieke M Aartsma-Rus, +2 more
1citations -
Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study2025 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery
Emma T. Groenwold, Alicia Montulet, Tiberiu Loredan Stan, Davy Van De Vijver, Diana McCorquodale, Nicholas McHugh, +7 more
2citations -
Additional file 4 of Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 function2023 Figshare dataset Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access
Melita Kaltak, Rocío Blanco-Garavito, Laurie L. Molday, Claire‐Marie Dhaenens, Eric E. Souied, Gerard J. Platenburg, +3 more
0citations