Research output per year
Research output per year
Personal profile
Research interests
Niek van Til has over 20 years of experience in the gene therapy field, currently focussing on preclinical development of tailor-made gene and cell therapies for leukodystrophies at the Amsterdam Leukodystrophy Center. The pipelines include the development of adeno-associated virus (AAV) vectors to deliver genetic therapeutics to the central nervous system for treatment of the leukodystrophy vanishing white matter. In addition, ex vivo hematopoietic stem cell gene therapy using lentiviral vectors are investigated for other leukodystrophies in which cross-correction is a mechanism of action. Other groundbreaking technologies, such as precise genome editing are also explored to treat these debilitating genetic diseases for which there is currently no cure.
Specialisation
Adeno associated virus gene therapy, preclinical studies, efficacy and safety testing, hematopoietic stem cell gene therapy, genome editing, metabolic diseases and leukodystrophies.
Related documents
Education/Academic qualification
PhD, Lentiviral gene therapy for the treatment of inherited liver disease, Amsterdam UMC - University of Amsterdam
Award Date: 28 Jun 2007
Master, Biology, Leiden University, Leiden, Netherlands
Award Date: 27 Feb 2001
Postdoctoral researcher/project leader, UMC Utrecht, Utrecht, Netherlands
2014 → 2021
Postdoctoral researcher, Erasmus Universiteit Rotterdam: Erasmus Medisch Centrum
2006 → 2014
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Research output
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In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice
Böck, D., Revers, I. M., Bomhof, A. S. J., Hillen, A. E. J., Boeijink, C., Kissling, L., Egli, S., Moreno-Mateos, M. A., van der Knaap, M. S., van Til, N. P. & Schwank, G., 1 May 2024, In: Molecular therapy. 32, 5, p. 1328-1343 16 p.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile43 Downloads (Pure) -
Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations
Yoon, J. K., Schindler, J. W., Loperfido, M., Baricordi, C., DeAndrade, M. P., Jacobs, M. E., Treleaven, C., Plasschaert, R. N., Yan, A., Barese, C. N., Dogan, Y., Chen, V. P., Fiorini, C., Hull, F., Barbarossa, L., Unnisa, Z., Ivanov, D., Kutner, R. H., Guda, S. & Oborski, C. & 8 others, , 6 Nov 2024, In: Molecular therapy. 32, 11, p. 3847-3864 18 p.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile34 Downloads (Pure) -
Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease
Dogan, Y., Barese, C. N., Schindler, J. W., Yoon, J. K., Unnisa, Z., Guda, S., Jacobs, M. E., Oborski, C., Maiwald, T., Clarke, D. L., Schambach, A., Pfeifer, R., Harper, C., Mason, C. & van Til, N. P., 8 Dec 2022, In: Molecular therapy. Methods & clinical development. 27, p. 464-487 24 p.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile13 Downloads (Pure) -
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease
Liang, Q., Vlaar, E. C., Catalano, F., Pijnenburg, J. M., Stok, M., van Helsdingen, Y., Vulto, A. G., Unger, W. W. J., van der Ploeg, A. T., Pijnappel, W. W. M. P. & van Til, N. P., 9 Jun 2022, In: Molecular therapy. Methods & clinical development. 25, p. 520-532 13 p.Research output: Contribution to journal › Article › Academic › peer-review
Open AccessFile13 Downloads (Pure) -
Gene and Cellular Therapies for Leukodystrophies
Aerts-Kaya, F. & van Til, N. P., 24 Oct 2023, In: Pharmaceutics. 15, 11, 2522.Research output: Contribution to journal › Review article › Academic › peer-review
Open AccessFile24 Downloads (Pure)