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A small-molecule stabilizer of the calpastatin-calpain-2 complex restores mitochondrial function and mitigates neurodegeneration - PubMed

4 hours ago
  • #calpain
  • #neurodegeneration
  • #mitochondria
  • A36, derived from CHIR99021, is a brain-penetrant small molecule that stabilizes the CAST-calpain-2 complex, limiting calpain-2 activation and mitochondrial damage.
  • In HD and tauopathy models, A36 normalized mitochondrial function, reduced oxidative stress, and improved neuron survival.
  • In vivo studies in R6/2 HD and PS19 tauopathy mice showed A36 reduced neurodegeneration, protein aggregation, and behavioral deficits.
  • Pharmacological stabilization of the CAST-calpain-2 complex is proposed as a therapeutic strategy for neurodegenerative diseases.