Specialization
Focus of research
Preclinical development of tailor-made gene and cell therapies for leukodystrophies.
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Draining the brain: Gene therapy reverses brain edema in mice with the leukodystrophy MLC
Min, R., van Til, N. P. & van der Knaap, M. S., 7 May 2025, In: Molecular Therapy. 33, 5, p. 1872-1873 2 p.Research output: Contribution to journal › Comment/Letter to the editor › Academic
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Assessment of non-myelotoxic agents as a preparatory regimen for hematopoietic stem cell gene therapy
Şeker, M. E., Erol, Ö. D., Pervin, B., Wagemaker, G., van Til, N. P. & Aerts-Kaya, F., 1 Jan 2025, In: Human Cell. 38, 1, 9.Research output: Contribution to journal › Article › Academic › peer-review
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Gene Therapy for Inherited Liver Disease: To Add or to Edit
Chen, Y., van Til, N. P. & Bosma, P. J., 1 Dec 2024, In: International journal of molecular sciences. 25, 23, p. 1-19 19 p., 12514.Research output: Contribution to journal › Review article › Academic › peer-review
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