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Gene Therapy for Inherited Liver Disease: To Add or to Edit
van Til, N. P., 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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Human post-mortem organotypic brain slice cultures: a tool to study pathomechanisms and test therapies
Plug, B. C., Revers, I. M., Breur, M., González, G. M., Timmerman, J. A., Meijns, N. R. C., Hamberg, D., Wagendorp, J., Wolf, N. I., Luchicchi, A., Mansvelder, H. D., van Til, N. P., van der Knaap, M. S. & Bugiani, M., Dec 2024, In: Acta Neuropathologica Communications. 12, 1, p. 1-17 17 p., 83.Research output: Contribution to journal › Article* › Academic › peer-review
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Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations
Treleaven, C., Chen, V. P., Ivanov, D., Kutner, R. H., Michaud, V. & van Til, N. P., 6 Nov 2024, In: Molecular therapy. 32, 11, p. 3847-3864 18 p.Research output: Contribution to journal › Article* › Academic › peer-review
