Gerard J. Platenburg
Leiden, ZH, Netherlands
Last Updated: 09/26/2026
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Found 15 publications
Cholesterol Conjugation Strategies to Enhance In Vivo Antisense Oligonucleotide-Mediated Exon Skipping in the Mouse Retina.
Journal: Journal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and Therapeutics
Published: July 20, 2026
ADAR RNA editing for cardiovascular disease: Targeting B4GALT1 to modulate lipid metabolism through reduced galactosyltransferase activity.
Journal: Molecular therapy. Nucleic acids
Published: October 30, 2025
Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 function.
Journal: Journal of translational medicine
Published: April 05, 2023
Targeted pseudouridylation: An approach for suppressing nonsense mutations in disease genes.
Journal: Molecular cell
Published: November 25, 2022
Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C.
Journal: Molecular therapy. Nucleic acids
Published: October 11, 2022
Intranasal administration of olanzapine has beneficial outcome in a rat activity-based anorexia model.
Journal: European neuropsychopharmacology : the journal of the European College of Neuropsychopharmacology
Published: October 05, 2022
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations.
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: February 12, 2021
QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study.
Journal: The Journal of investigative dermatology
Published: November 07, 2019
Last Updated: 09/26/2026