Marieke E. Willemse
Nijmegen, GE 6525GA, Netherlands
Last Updated: 09/26/2026
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Found 21 publications
CRISPR/dCas9-mediated tuning of DMPK transcription reveals a quantitative relationship between toxic repeat RNA expression and MBNL1 activity in myotonic dystrophy.
Journal: Human molecular genetics
Published: May 15, 2026
Generation of isogenic rescue iPSC lines by targeted CTG-repeat excision for myotonic dystrophy type 1.
Journal: Stem cell research
Published: April 29, 2026
Population Pharmacokinetics of Bosutinib in Children with Philadelphia Chromosome-Positive Chronic Myelogenous Leukemia Based on a Phase I Dose-Finding Trial (ITCC-054/COG AAML1921) and Pediatric Recommended Phase II Dosage Evaluation.
Journal: Clinical pharmacokinetics
Published: February 05, 2026
Repeat length as a key determinant for disease severity and antisense oligonucleotide activity in myotonic dystrophy type 1.
Journal: Molecular therapy. Methods & clinical development
Published: January 31, 2025
Ameliorated cellular hallmarks of myotonic dystrophy in hybrid myotubes from patient and unaffected donor cells.
Journal: Stem cell research & therapy
Published: July 05, 2024
Bosutinib in Resistant and Intolerant Pediatric Patients With Chronic Phase Chronic Myeloid Leukemia: Results From the Phase I Part of Study ITCC054/COG AAML1921.
Journal: Journal of clinical oncology : official journal of the American Society of Clinical Oncology
Published: November 30, 2023
The PedAL/EuPAL Project: A Global Initiative to Address the Unmet Medical Needs of Pediatric Patients with Relapsed or Refractory Acute Myeloid Leukemia.
Journal: Cancers
Published: November 20, 2023
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1.
Journal: Molecular therapy. Nucleic acids
Published: October 26, 2022
Recovery in the Myogenic Program of Congenital Myotonic Dystrophy Myoblasts after Excision of the Expanded (CTG)n Repeat.
Journal: International journal of molecular sciences
Published: October 30, 2019
Intrinsic Myogenic Potential of Skeletal Muscle-Derived Pericytes from Patients with Myotonic Dystrophy Type 1.
Journal: Molecular therapy. Methods & clinical development
Published: June 06, 2019
(CTG)n repeat-mediated dysregulation of MBNL1 and MBNL2 expression during myogenesis in DM1 occurs already at the myoblast stage.
Journal: PloS one
Published: February 13, 2019
Super-Resolution Correlative Light and Electron Microscopy (SR-CLEM) Reveals Novel Ultrastructural Insights Into Dendritic Cell Podosomes.
Journal: Frontiers in immunology
Published: May 30, 2018
Last Updated: 09/26/2026