A framework for individualized splice-switching oligonucleotide therapy

• Safety and toxicity assessment of antisense oligonucleotides in brain-relevant human cellular models

  2026 Molecular Therapy — Nucleic Acids article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  0

  citations
• Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies

  2026 Orphanet Journal of Rare Diseases article Economics, Econometrics and Finance Pharmaceutical Economics and Policy Open access

  3

  citations
• A scalable and equitable framework for target and patient prioritisation in rare disease antisense therapeutics

  2026 medRxiv preprint Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access

  1

  citations
• Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study

  2025 Genetics in Medicine article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases

  4

  citations
• Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

  2025 Molecular Therapy — Nucleic Acids article Medicine Neurogenetic and Muscular Disorders Research Open access

  10

  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
• From roadmap to a sustainable end-to-end individualized therapy pathway

  2025 Therapeutic Advances in Rare Disease article Medicine Biomedical Ethics and Regulation Open access

  8

  citations
• Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments

  2025 The American Journal of Human Genetics article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access

  32

  citations
• The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap

  2024 Nature Reviews Drug Discovery review Medicine Biomedical Ethics and Regulation

  41

  citations
• Consensus guidelines for eligibility assessment of pathogenic variants to antisense oligonucleotide treatments

  2024 medRxiv preprint Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access

  5

  citations
• Advancing ASO therapies from development to implementation

  2024 Nature Medicine article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access

  7

  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
• 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
• Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders

  2024 Communications Medicine article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 5 patents

  272

  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
• Applying Lessons Learned from Developing Exon Skipping for Duchenne to Developing Individualized Exon Skipping Therapy for Patients with Neurodegenerative Diseases

  2023 Synlett article Medicine Neurogenetic and Muscular Disorders Research Open access

  2

  citations

16 results

• Whole‐genome sequencing holds the key to the success of gene‐targeted therapies

  2022 American Journal of Medical Genetics Part C Seminars in Medical Genetics article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access

  14

  citations

1 result