Overview on AON Design

• Expanding the options for therapeutic exon skipping as a future treatment for USH2A -associated disease by 3D structural modeling of newly formed hybrid domains

  2026 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders

  1

  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

  2

  citations
• Determining off-target effects of splice-switching antisense oligonucleotides using short read RNAseq in neuronally differentiated human induced pluripotent stem cells

  2025 Human Molecular Genetics article Biochemistry, Genetics and Molecular Biology RNA Research and Splicing Open access

  4

  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

  2

  citations
• The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing

  2025 Nucleic Acids Research article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  25

  citations
• Determining off-target effects of splice-switching antisense oligonucleotides using short read RNAseq in neuronally differentiated human induced pluripotent stem cells

  2025 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology RNA Research and Splicing Open access

  1

  citations
• Treatability of the KMT2-Associated Neurodevelopmental Disorders Using Antisense Oligonucleotide-Based Treatments

  2024 Human Mutation article Biochemistry, Genetics and Molecular Biology Genetics and Neurodevelopmental Disorders Open access

  3

  citations
• Antisense oligonucleotide-mediated terminal intron retention of endoglin: A potential strategy to inhibit renal interstitial fibrosis

  2024 Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease article Medicine Chronic Kidney Disease and Diabetes Open access

  4

  citations
• Practical Recommendations for the Selection of Patients for Individualized Splice-Switching ASO-Based Treatments

  2024 Human Mutation article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access

  7

  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

  5

  citations
• Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics

  2024 Therapeutic Advances in Rare Disease article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  9

  citations
• DMD antisense oligonucleotide mediated exon skipping efficiency correlates with flanking intron retention time and target position within the exon

  2023 RNA Biology article Biochemistry, Genetics and Molecular Biology Muscle Physiology and Disorders Open access

  2

  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

  94

  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

  55

  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

  83

  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

  13

  citations
• Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations

  2021 Molecular Therapy article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access cited by 8 patents

  148

  citations
• DNA glycosylase Neil3 regulates vascular smooth muscle cell biology during atherosclerosis development

  2021 Atherosclerosis article Immunology and Microbiology Atherosclerosis and Cardiovascular Diseases Open access

  19

  citations

18 results