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- Title
Allosteric Modulation of GSK-3β as a New Therapeutic Approach in Limb Girdle Muscular Dystrophy R1 Calpain 3-Related.
- Authors
Rico, Anabel; Guembelzu, Garazi; Palomo, Valle; Martínez, Ana; Aiastui, Ana; Casas-Fraile, Leire; Valls, Andrea; López de Munain, Adolfo; Sáenz, Amets
- Abstract
Limb-girdle muscular dystrophy R1 calpain 3-related (LGMDR1) is an autosomal recessive muscular dystrophy produced by mutations in the CAPN3 gene. It is a rare disease and there is no cure or treatment for the disease while the pathophysiological mechanism by which the absence of calpain 3 provokes the dystrophy in muscles is not clear. However, key proteins implicated in Wnt and mTOR signaling pathways, which regulate muscle homeostasis, showed a considerable reduction in their expression and in their phosphorylation in LGMDR1 patients' muscles. Finally, the administration of tideglusib and VP0.7, ATP non-competitive inhibitors of glycogen synthase kinase 3β (GSK-3β), restore the expression and phosphorylation of these proteins in LGMDR1 cells, opening the possibility of their use as therapeutic options.
- Subjects
CALPAIN; MUSCULAR dystrophy; THERAPEUTICS; LIMB-girdle muscular dystrophy; GLYCOGEN synthase kinase; WNT proteins
- Publication
International Journal of Molecular Sciences, 2021, Vol 22, Issue 14, p7367
- ISSN
1661-6596
- Publication type
Article
- DOI
10.3390/ijms22147367