Lenie Vanhove, Marthe Dias, Delphine De Coninck, Charlotte Grootaert, Andreja Rajkovic, Delfien Syx, Arnaud Vanlander, Jan L De Bleecker, Boel De Paepe
Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular disorder characterized by progressive muscle degeneration resulting in severe muscle weakness and motor function loss. Historically, therapeutic innovation has been facilitated by research in the mdx mouse, which is the most commonly used model for studying DMD. However, the inherent limitations of this model necessitate the formulation of alternative strategies. The present study proposes the zebrafish as an additional pre-clinical disease model in which to evaluate the benefits of nutraceuticals for DMD. The focus of this study will be taurine, a non-protein building amino acid that has emerged as a promising disease-modifying supplement for muscle wasting conditions, including aging-associated sarcopenia and DMD. The present study will evaluate the potential of early taurine supplementation to enhance the efficiency of autophagy and to improve mitochondrial health in a zebrafish DMD model. The study will facilitate a more rigorous examination of the claims pertaining to the beneficial effects of taurine on DMD progression, thereby informing the decision-making process regarding its potential implementation in human disease management.