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• The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

  2020 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access cited by 1 patent

  Annemieke M Aartsma-Rus, David R. Corey

  139

  citations
• Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

  2022 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 3 patents

  Aurélie Goyenvalle, Cecilia Jimenez-Mallebrera, Willeke van Roon‐Mom, Sabine Sewing, Arthur Μ. Krieg, Virginia Arechavala‐Gomeza, +1 more

  94

  citations
• Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives

  2021 Nucleic Acid Therapeutics article Medicine Biomedical Ethics and Regulation Open access

  Matthis Synofzik, Willeke van Roon‐Mom, Georg Marckmann, Hermine A. van Duyvenvoorde, Holm Graeßner, Rebecca Schüle, +1 more

  83

  citations
• Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides

  2022 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Research and Splicing Open access

  Annemieke M Aartsma-Rus, Alejandro Garanto, Willeke van Roon‐Mom, Erin M. McConnell, Victoria Suslovitch, Winston X. Yan, +2 more

  55

  citations
• From Failure to Meet the Clinical Endpoint to U.S. Food and Drug Administration Approval: 15th Antisense Oligonucleotide Therapy Approved Qalsody (Tofersen) for Treatment of SOD1 Mutated Amyotrophic Lateral Sclerosis

  2023 Nucleic Acid Therapeutics article Medicine Amyotrophic Lateral Sclerosis Research Open access

  Willeke van Roon‐Mom, Chantal M. Ferguson, Annemieke M Aartsma-Rus

  52

  citations
• Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy

  2023 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

  19

  citations
• OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides

  2023 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  Madeleine Antonsson, Sándor Bátkai, Sieto Bosgra, Girish Rajmal Chopda, Wouter H. P. Driessen, Jeffrey W.-D. Foy, +8 more

  18

  citations
• 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 Dystrophy

  2023 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

  16

  citations
• Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy

  2022 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  Sarah Engelbeen, Svetlana Pasteuning-Vuhman, Rubina Giare Parmar, Klaus Charissé, Laura Sepp‐Lorenzino, Muthiah A. Manoharan, +2 more

  14

  citations
• Antisense Oligonucleotide-Induced Amyloid Precursor Protein Splicing Modulation as a Therapeutic Approach for Dutch-Type Cerebral Amyloid Angiopathy

  2021 Nucleic Acid Therapeutics article Medicine Alzheimer's disease research and treatments Open access

  Elena Daoutsali, Tsinatkeab T. Hailu, Ronald A.M. Buijsen, Barry A. Pepers, Linda M. van der Graaf, Marcel M. Verbeek, +3 more

  13

  citations
• The Role of Patient Involvement When Developing Therapies

  2021 Nucleic Acid Therapeutics article Health Professions Mental Health and Patient Involvement Open access

  Annemieke M Aartsma-Rus, Elizabeth Vroom, Daniel O’Reilly

  8

  citations
• Report of the European Medicines Agency Conference on RNA-Based Medicines

  2024 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  Falk Ehmann, Andreas N. Kuhn, Anna M.G. Pasmooij, Arjon van Hengel, Brigitte Anliker, Daniel C. Capaldi, +19 more

  7

  citations
• Highly Potent Antisense Oligonucleotides Locked Nucleic Acid Gapmers Targeting the SARS-CoV-2 RNA Genome

  2023 Nucleic Acid Therapeutics article Medicine SARS-CoV-2 and COVID-19 Research Open access

  Vita Dauksaite, Ali Taş, Falk Wachowius, Martijn J. van Hemert, Eric J. Snijder, Eric P. van der Veer, +1 more

  6

  citations
• Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice

  2024 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  Mick M. Welling, Ernst Suidgeest, Esmée Nieuwenhuize, Thomas de Vlaam, Tsinatkeab T. Hailu, Louise van der Weerd, +1 more

  5

  citations
• Opportunities for More Tailored Approaches to Genotoxicity Testing and Carcinogenicity Strategy for Oligonucleotide Therapeutics: Outcome of an Industry Survey

  2025 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology Carcinogens and Genotoxicity Assessment Open access

  Joel D. Parry, Tod A. Harper, Patrik Urban Andersson, Joanne M. Elloway, Natalie S. Holman, William E. Achanzar, +10 more

  3

  citations
• A Guide to Chemical Considerations for the Pre-Clinical Development of Oligonucleotides

  2024 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  Daniel O’Reilly, Willeke van Roon‐Mom, Annemieke M Aartsma-Rus

  2

  citations
• Design and In Vitro Evaluation of a Novel Antisense Oligonucleotide for Treatment of BK Virus Infection

  2025 Nucleic Acid Therapeutics article Medicine Polyomavirus and related diseases Open access

  Jurriën Prins, Janneke Kouwenberg, Lizanne Daleman, Iris van Wissen, Bianca Matthee, Angela Koudijs, +15 more

  2

  citations
• Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study

  2025 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

  2

  citations
• 17th Annual Meeting of the Oligonucleotide Therapeutics Society: A Tribute to Bob Letsinger, Progress in N = 1 Treatments, and Successful First In-Humans CRISPR Trials

  2022 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery

  Ronald A.M. Buijsen, Aurélie Lacroix, Joseph Ochaba, Daniel O’Reilly, Leonora Abdullahu, Erin M. McConnell

  0

  citations
• In Vivo Phage Display for the Identification of Muscle Homing Peptides to Improve the Delivery of Phosphorodiamidate Morpholino Oligomers for Duchenne Muscular Dystrophy Therapy

  2025 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  Anne-Fleur E. Schneider, Christa L. Tanganyika-de Winter, Hailiang Mei, Silvana M.G. Jirka, Xuyu Tan, Emily G. Thompson, +4 more

  0

  citations

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