Several studies have implicated lysosomal and mitochondrial dysfunction in the pathophysiology of neurodegenerative disorders such as Alzheimer’s and Parkinson’s diseases. Scientists at the Icahn School of Medicine, Mount Sinai, have reported a novel strategy to rectify lysosomal build-up in lysosomal storage disorders that can potentially bypass the existing genetic defect. They demonstrated that activation of the mitochondrial chaperone tumor necrosis factor receptor-associated protein 1 (TRAP 1) corrects the lysosomal storage phenotype in cells isolated from patients suffering from multiple lysosomal storage disorders , including Niemann–Pick C1.
Study findings Small molecules ML405 and 1685, TRAP1 agonists, correct the lipid storage in LSDs, including NPC1 TRAP1 agonists ameliorate mitochondrial stress and improve lysosomal function. Mechanism: TRAP1 activation inhibits oxidative…
