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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 -
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 -
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 -
Sequence determinants of efficient exon 44 skipping in Duchenne muscular dystrophy define design principles for steric-blocking antisense oligonucleotides2026 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access
Enyang Han, Katherine Webster, Tiberiu Loredan Stan, C. Tanganyika-de Winter, Elizabeth M. van der Pijl, Jordan Tahquechi, +7 more
0citations -
Consensus guidelines for eligibility assessment of pathogenic variants to antisense oligonucleotide treatments2024 medRxiv preprint Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access
David Cheerie, Margaret M. Meserve, Danique Beijer, Charu Kaiwar, Logan Newton, Ana Lisa Taylor Tavares, +31 more
5citations -
Antisense oligonucleotide therapy for the common Stargardt disease type 1-causing variant in ABCA42022 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access
Melita Kaltak, Davide Piccolo, Sang‐Eun Lee, Kalyan Dulla, Thomas Hoogenboezem, Wouter Beumer, +5 more
1citations -
Clinical sequencing identifies potential actionable alterations in a high rate of urachal and primary bladder adenocarcinomas2022 Research Square preprint Biochemistry, Genetics and Molecular Biology Cancer Genomics and Diagnostics Open access
Melinda Váradi, Nikolett T. Nagy, Henning Reis, Boris Alexander Hadaschik, Christian Niedworok, Orsolya Módos, +20 more
2citations
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