Developing juvenile zebrafish as a preclinical model for therapeutic innovation in muscular dystrophyDuchenne
Byttebier Ella, 2025
Duchenne Muscular Dystrophy (DMD) is a severe, progressive neuromuscular disorder for which no curative treatment exists. Gene therapies and exon-skipping strategies targeting the DMD gene have entered the clinic, however, their efficacy is limited by mutation heterogeneity necessitating highly personalized, mutation-specific approaches. Glucocorticosteroids remain the standard of care but are associated with significant side effects. As current molecular therapies do not cure this fatal disease, there is a pressing need for supportive interventions that improve patients comfort and quality of life and can be safely combined with existing treatments.
Interest in supplements among DMD patients is increasing, driven by their wide availability and the perception that they are safe. However, most supplements lack rigorous preclinical or clinical evaluation, and their efficacy and safety in the context of DMD remain largely unproven. The assumption that naturally derived compounds cannot harm is misleading, as supplements can cause adverse effects or interact negatively with conventional therapies. Furthermore, healthcare professionals often do not proactively address supplement use with patients, contributing to a gap in informed clinical decision-making1.
This study aims to establish and validate the sapje zebrafish as a preclinical model to evaluate nutraceuticals previously shown to be beneficial in the mdx mouse. Unlike mdx mice, sapje zebrafish exhibit a phenotype more reflective of human DMD. Developing a relevant model is essential for generating reliable data on the therapeutic potential and safety of these compounds. The application of this research will support evidence-based guidance for clinicians and patients regarding the use of nutraceuticals in DMD management.
| Promotor | Boel De Paepe |
| Opleiding | Biomedische Wetenschappen |
| Kernwoorden | Duchenne muscular dystrophy sapje zebrafish Dystrophin |