| Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders MC Lauffer, W van Roon-Mom, A Aartsma-Rus, N= 1 Collaborative Communications Medicine 4 (1), 6, 2024 | 85 | 2024 |
| An ancestral 10-bp repeat expansion in VWA1 causes recessive hereditary motor neuropathy AT Pagnamenta, R Kaiyrzhanov, Y Zou, SI Da'as, R Maroofian, ... Brain 144 (2), 584-600, 2021 | 30 | 2021 |
| The basic helix‐loop‐helix transcription factor TCF4 impacts brain architecture as well as neuronal morphology and differentiation M Schoof, M Hellwig, L Harrison, D Holdhof, MC Lauffer, J Niesen, S Virdi, ... European Journal of Neuroscience 51 (11), 2219-2235, 2020 | 28 | 2020 |
| TCF4 (E2-2) harbors tumor suppressive functions in SHH medulloblastoma M Hellwig, MC Lauffer, M Bockmayr, M Spohn, DJ Merk, L Harrison, ... Acta neuropathologica 137, 657-673, 2019 | 22 | 2019 |
| Development of tailored splice-switching oligonucleotides for progressive brain disorders in Europe: development, regulation, and implementation considerations A Aartsma-Rus, W van Roon-Mom, M Lauffer, C Siezen, B Duijndam, ... Rna 29 (4), 446-454, 2023 | 21 | 2023 |
| N= 1 Collaborative (2024). Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders MC Lauffer, W van Roon-Mom, A Aartsma-Rus Commun. Med 4 (6), 0 | 5 | |
| Practical Recommendations for the Selection of Patients for Individualized Splice‐Switching ASO‐Based Treatments B Zardetto, MC Lauffer, W van Roon-Mom, A Aartsma-Rus Human Mutation 2024 (1), 9920230, 2024 | 3 | 2024 |
| Consensus guidelines for eligibility assessment of pathogenic variants to antisense oligonucleotide treatments D Cheerie, M Meserve, D Beijer, C Kaiwar, L Newton, ALT Tavares, ... medRxiv, 2024.09. 27.24314122, 2024 | 2 | 2024 |
| Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics A Aartsma-Rus, RWJ Collin, Y Elgersma, MC Lauffer, W van Roon-Mom Therapeutic Advances in Rare Disease 5, 26330040241273465, 2024 | 2 | 2024 |
| Organ-on-a-chip technologies to study neuromuscular disorders: possibilities, limitations, and future hopes MC Lauffer Medizinische Genetik 33 (3), 261-267, 2021 | 2 | 2021 |
| Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: a retrospective cohort study D Cheerie, MC Lauffer, L Newton, K Amburgey, D Beijer, B Haque, ... medRxiv, 2025.02. 10.25321921, 2025 | | 2025 |
| ExonViz: A website and Python package to visualize transcripts and genetic variants RR van den Berg, MC Lauffer, JFJ Laros medRxiv, 2024.09. 18.24313945, 2024 | | 2024 |
| Treatability of the KMT2‐Associated Neurodevelopmental Disorders Using Antisense Oligonucleotide‐Based Treatments B Zardetto, W van Roon-Mom, A Aartsma-Rus, MC Lauffer Human Mutation 2024 (1), 9933129, 2024 | | 2024 |
| P171: Developing an approach to screening rare genetic diagnoses for amenability to bespoke genetic therapy development D Cheerie, M Lauffer, D Beijer, M Synofzik, A Aartsma-Rus, M Szego, ... Genetics in Medicine Open 2, 101068, 2024 | | 2024 |
| Patient and variant stratification for personalized genetic treatments of nano-rare diseases MC Lauffer, B Zardetto, W van Roon-Mom, A Aartsma-Rus EUROPEAN JOURNAL OF HUMAN GENETICS 32, 737-738, 2024 | | 2024 |
| 1 Mutation 1 Medicine: a European platform for ASO development and treatments for individuals with severely debilitating or life threatening nano-rare neurological diseases H Graessner, M Synofzik, A Aartsma-Rus, W van Roon-Mom, M Lauffer, ... EUROPEAN JOURNAL OF HUMAN GENETICS 32, 735-735, 2024 | | 2024 |
| The Dutch Center for RNA Therapeutics: a center to develop antisense oligonucleotide therapies for patients with nano-rare mutations MC Lauffer, A Aartsma-Rus, RWJ Collin, Y Elgersma, W van Roon-Mom EUROPEAN JOURNAL OF HUMAN GENETICS 31, 665-665, 2023 | | 2023 |
| The Dutch Center of RNA Therapeutics: developing mutation-specific antisense oligonucleotide therapies for patients with eye and brain diseases carrying unique mutations A Aartsma-Rus, M Lauffer, R Collin, Y Elgersma, W van Roon-Mom FEBS OPEN BIO 12, 63-63, 2022 | | 2022 |
| The role of TCF4 in cerebellum development and its influence on formation and growth of medulloblastoma MC Lauffer lmu, 2019 | | 2019 |
| Evidence for TCF4 playing a role as tumor suppressor in medulloblastoma M Lauffer, M Hellwig, U Schüller Klinische Pädiatrie 229 (03), A25, 2017 | | 2017 |
