ProQR Therapeutics (Netherlands)

Recent papers from ProQR Therapeutics (Netherlands)

1. ADAR RNA editing for cardiovascular disease: Targeting B4GALT1 to modulate lipid metabolism through reduced galactosyltransferase activity.2026
2. Cholesterol Conjugation Strategies to Enhance In Vivo Antisense Oligonucleotide-Mediated Exon Skipping in the Mouse RetinaJournal of Ocular Pharmacology and Therapeutics · 2026
3. ADAR RNA editing for cardiovascular disease: Targeting B4GALT1 to modulate lipid metabolism through reduced galactosyltransferase activityMolecular Therapy — Nucleic Acids · 2026
4. Therapeutic rescue of pathogenic asparaginyl-tRNA synthetase allelesMolecular Therapy — Nucleic Acids · 2026
5. Zigakibart Potently Inhibits A Proliferation-Inducing Ligand-Mediated IgA Production, Addressing IgAN Pathogenesis Root CausesKidney International Reports · 2026
6. Adamts5 deletion exacerbates inflammation and fibrosis resulting in compromised skeletal muscle regenerationAJP Cell Physiology · 2026
7. Evaluation of in vitro liver models and non-human primates for predicting ADAR RNA editing efficiency in human liverScientific Reports · 2025
8. Serum Alpha-Linolenic Acid and Long-Term Multiple Sclerosis Activity and ProgressionNeurology · 2025
9. ADAMTS-5 inhibition reduces muscle inflammation and fibrosis and improves function in mouse models of Duchenne muscular dystrophyScience Translational Medicine · 2025
10. Accurate Quantification of Mutant and Wild-Type polyQ Proteins Using Simple Western Capillary ImmunoassaysMolecular Neurobiology · 2025
11. Impact of mesenchymal stromal/stem cell infusions on circulating inflammatory biomarkers in COVID-19 patients: analysis of a phase I-IIa trialCytotherapy · 2025
12. Opportunities for More Tailored Approaches to Genotoxicity Testing and Carcinogenicity Strategy for Oligonucleotide Therapeutics: Outcome of an Industry SurveyNucleic Acid Therapeutics · 2025
13. RNA‐Guided RNA Pseudouridylation and 2’‐O‐MethylationIsrael Journal of Chemistry · 2024
14. High Levels of Mutant Huntingtin Protein in Tear Fluid From Huntington’s Disease Gene Expansion CarriersJournal of Movement Disorders · 2024
15. Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in MiceNucleic Acid Therapeutics · 2024
16. QR-1011 restores defective ABCA4 splicing caused by multiple severe ABCA4 variants underlying Stargardt diseaseScientific Reports · 2024
17. Pseudouridylation-mediated gene expression modulationBiochemical Journal · 2024
18. OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic OligonucleotidesNucleic Acid Therapeutics · 2023
19. Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 functionJournal of Translational Medicine · 2023
20. Stargardt disease-associated missense and synonymous ABCA4 variants result in aberrant splicingHuman Molecular Genetics · 2023