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isolecanoric acid
- Development stage
- Preclinical
- Modality
- Small Molecules
01
Overview
Isolecanoric acid (ILA) is a natural product, specifically a depside isolated from fungal cultures, that functions as a potent inhibitor of Glycogen Synthase Kinase-3 beta (GSK-3β). It is primarily being researched for the treatment of Duchenne muscular dystrophy (DMD), where it aims to address muscle degeneration and chronic inflammation. By inhibiting GSK-3β, ILA stabilizes β-Catenin, thereby promoting the myogenic differentiation of muscle stem cells and myoblasts. Preclinical evidence suggests that ILA treatment improves muscle regeneration, functional performance, and reduces fibrosis by modulating the inflammatory response in macrophages.
- Other names
- ILA
02
Targets
GSK3 (Glycogen synthase kinase 3 beta)
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