Niek van Til

Assistant professor

Amsterdam UMC Locatie VUmc

Netherlands

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About

Niek van Til is an Assistant Professor at Amsterdam UMC Locatie VUmc in the Netherlands. His research focuses on gene therapy approaches for various inherited diseases, including leukodystrophies and liver diseases. Recent work includes studies on CRISPR-Cas9 applications and hematopoietic stem cell gene therapy. His contributions significantly advance the understanding and treatment of genetic disorders through innovative therapeutic strategies.

Articles (19)

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2025

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2020

Collaborators (2)

Huib Mansvelder

University of Amsterdam

NETHERLANDS

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Gerald Schwank

Associate Professor

University of Zurich

SWITZERLAND

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Niek van Til

Research Interests

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About

Articles (19)

Draining the brain: Gene therapy reverses brain edema in mice with the leukodystrophy MLC

Human post-mortem organotypic brain slice cultures: a tool to study pathomechanisms and test therapies.

Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AML.

In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice.

Gene Therapy for Inherited Liver Disease: To Add or to Edit

Assessment of non-myelotoxic agents as a preparatory regimen for hematopoietic stem cell gene therapy

Correction of Griscelli Syndrome Type 2 causing mutations in the RAB27A gene with CRISPR/Cas9

Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations

Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.

Lentiviral gene therapy with IGF2-tagged GAA normalizes the skeletal muscle proteome in murine Pompe disease.

Gene and Cellular Therapies for Leukodystrophies

Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease.

IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy.

High-throughput characterization of hematopoietic stem cell engraftment after intravenous and intracerebroventricular dosing

Lentiviral Gene Therapy Prevents Anti-Human Acid α-Glucosidase Antibody Formation in Murine Pompe Disease

Gene Therapy Developments for Pompe Disease

Efficient lentiviral transduction method to gene modify cord blood CD8<sup>+</sup> T cells for cancer therapy applications.

Gene Augmentation and Editing to Improve TCR Engineered T Cell Therapy against Solid Tumors

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease.

Collaborators (2)

Huib Mansvelder

Gerald Schwank

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