Glucocorticoid receptors are locally useful in DMD

American researchers sought to better understand the function and effects of long-term corticosteroid therapy in Duchenne muscular dystrophy (DMD):

• the gene encoding the glucocorticoid receptor was invalidated in one of the animal models of DMD (the mdx52 mouse),
• the double knock-outTechnique permettant d’invalider un gène. mice thus created were studied from a physiological (strength and function measurements) and histological (skeletal muscle fibres and cardiomyocytesCellules musculaires cardiaques.) point of view,
• the phenotype obtained was much more severe than that of the original mouse.

This work argues in favour of a protective effect of this receptor in the physiological state but also in the context of DMD, as observed in clinical trials.

The glucocorticoid receptor acts locally to protect dystrophic muscle and heart during disease. Oliver T, Nguyen NY, Tully CB et al. Dis Model Mech. 2024 May 1;17(5):dmm050397.