Niek P. Van Til
Amsterdam, NH, Netherlands
Last Updated: 09/26/2026
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Found 37 publications
Assessment of Potential Side Effects Related To RAB27A Gene Therapy in Stem Cells.
Journal: Stem cell reviews and reports
Published: August 11, 2025
Draining the brain: Gene therapy reverses brain edema in mice with the leukodystrophy MLC.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: March 31, 2025
Gene Therapy for Inherited Liver Disease: To Add or to Edit.
Journal: International journal of molecular sciences
Published: August 23, 2024
Combining CRISPR-Cas9 and TCR exchange to generate a safe and efficient cord blood-derived T cell product for pediatric relapsed AML.
Journal: Journal for immunotherapy of cancer
Published: March 18, 2024
Human post-mortem organotypic brain slice cultures: a tool to study pathomechanisms and test therapies.
Journal: Acta neuropathologica communications
Published: February 13, 2024
Correction of Griscelli Syndrome Type 2 causing mutations in the RAB27A gene with CRISPR/Cas9.
Journal: Turkish journal of biology = Turk biyoloji dergisi
Published: December 01, 2023
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: November 09, 2023
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: October 17, 2023
Gene and Cellular Therapies for Leukodystrophies.
Journal: Pharmaceutics
Published: September 17, 2023
Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Journal: Annals of clinical and translational neurology
Published: January 14, 2023
Last Updated: 09/26/2026