A xenotransplantation model for reactivation of paternal UBE3A using human-specific antisense oligonucleotides

a-xenotransplantation-model-for-reactivation-of-paternal-ube3a-using-human-specific-antisense-oligonucleotides
A xenotransplantation model for reactivation of paternal UBE3A using human-specific antisense oligonucleotides

Data availability

Data generated and analyzed in this study may be requested from the corresponding author, upon reasonable request.

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Acknowledgements

We would like to thank Roche for providing the ASOs used in this study. Roche did not participate in study design or analysis. We would also like to thank Claudia Milazzo (Erasmus MC) for technical assistance.

Funding

This work was supported by funding from the Netherlands Organ-on-Chip Initiative, an NWO Gravitation project (024.003.001), from the Ministry of Education, Culture and Science of the government of the Netherlands (to S.A.K., and F.M.S.d.V.), an NWO ZonMw Rubicon project (452022211, to B.L.) and by the TAILORED project of the ZonMw PSIDER program (10250022110002, to S.A.K., F.M.S.d.V., Y.E., M.G.B., H.S.).

Author information

Author notes

  1. Hilde Smeenk and Bas Lendemeijer contributed equally to this work. Femke M.S. de Vrij and Steven A. Kushner jointly supervised this work.

Authors and Affiliations

  1. Department of Psychiatry, Erasmus MC University Medical Center, Rotterdam, The Netherlands

    Hilde Smeenk, Bas Lendemeijer, Mirle G. Buurma, Michell A. Forgione, Denise E. Slump, Witte J. G. Hoogendijk & Femke M. S. de Vrij

  2. Stavros Niarchos Foundation (SNF) Center for Precision Psychiatry & Mental Health, Columbia University, New York, NY, USA

    Bas Lendemeijer & Steven A. Kushner

  3. Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands

    Roos A. Monshouwer, Ilse Wallaard, Edwin J. Mientjes & Ype Elgersma

  4. Erasmus MC Expertise Center for Neurodevelopmental Disorders (ENCORE), Erasmus MC University Medical Center, Rotterdam, The Netherlands

    Edwin J. Mientjes, Ype Elgersma & Femke M. S. de Vrij

  5. Department of Psychiatry, Columbia University Irving Medical Center, New York, NY, USA

    Steven A. Kushner

Authors

  1. Hilde Smeenk
  2. Bas Lendemeijer
  3. Mirle G. Buurma
  4. Michell A. Forgione
  5. Denise E. Slump
  6. Roos A. Monshouwer
  7. Ilse Wallaard
  8. Edwin J. Mientjes
  9. Witte J. G. Hoogendijk
  10. Ype Elgersma
  11. Femke M. S. de Vrij
  12. Steven A. Kushner

Contributions

The author contributions were as follows: experimental design: H.S., B.L., I.W., E.J.M, Y.E., F.M.S.d.V., S.A.K; data acquisition: H.S., B.L., M.G.B., M.A.F., R.A.M., D.E.S.; data analysis: H.S., B.L; writing the manuscript: H.S., B.L., F.M.S.d.V., S.A.K.; review of manuscript: all authors; supervision: E.J.M., W.J.G.H., Y.E., F.M.S.d.V., S.A.K.; H.S. and B.L. contributed equally to the manuscript; F.M.S.d.V. and S.A.K. contributed equally to the manuscript.

Corresponding authors

Correspondence to Femke M. S. de Vrij or Steven A. Kushner.

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Competing interests

The authors declare no competing interests.

Ethical approval

All methods were performed in accordance with the relevant guidelines and regulations. In addition, all mouse experiments were conducted in accordance with ARRIVE guidelines and the European Commission Council Directive 2010/63/EU. We obtained national and local approval under CCD license AVD1010020173544 and AVD10100202215931 (Centrale Commissie Dierproeven, the Hague, the Netherlands).

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Smeenk, H., Lendemeijer, B., Buurma, M.G. et al. A xenotransplantation model for reactivation of paternal UBE3A using human-specific antisense oligonucleotides. Sci Rep (2026). https://doi.org/10.1038/s41598-026-41197-9

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  • DOI: https://doi.org/10.1038/s41598-026-41197-9