Topic: lysosome

The aim of this study was to investigate the ability of GT-02287 to rescue nest-building behaviour following delayed administration in a mouse model of GBA1-Parkinson's disease (GBA1-PD). Nest building is a non-invasive test to study activities of daily living and cognitive performance in rodent models. GT-02287 is an orally bioavailable, brain penetrant, small-molecule structurally targeted allosteric regulator that binds to glucocerebrosidase (GCase), stabilizes it, and increases its function by chaperoning it to the lysosome. It is currently in Phase 1 clinical development for the treatmen

In this study we investigated whether the presence of mutations in TMEM175 gene could reflect a characteristic lipidomic, metabolomic and proteomic signature of PD patients both at circulating and cellular levels.The study cohort included two groups of patients that differ for the presence of TMEM175 mutations, and a group of matched controls. We adopted an untargeted omics approach to disclose lipidomic, metabolomic and proteomic alterations in PD patients carrying TMEM175 mutations both in plasma and dermal fibroblasts.Integrated analysis of omics data revealed wide deregulation of lysosome,