| Gene Symbol | ent-gene-304eb5d4 |
| Model | iPSC-derived microglia + dopaminergic neurons from PD patients (LRRK2 G2019S, idiopathic) and healthy controls |
| Condition Groups | Control, PD neurons only, PD neurons + NLRP3 activators (ATP, nigericin), PD neurons + NLRP3 inhibitors (MCC950, VX-765) |
| Readouts | - Behavioral: cylinder test, stepping test, gait analysis - Biochemical: striatal dopamine, TH+ neuron counts in SNc - Molecular: NLRP3 pathway activation in microglia - Inflammation: Iba1+ microglial density, cytokine levels |
| Models | MPTP-induced PD model, α-synuclein pre-formed fibril (PFF) model |
| Treatment Groups | Vehicle, NLRP3 inhibitor (MCC950, 10mg/kg i.p. daily), Positive control (L-DOPA) |
| Duration | 52 weeks |
| Cohort | 300 PD patients (various stages), 100 controls |
| Samples | CSF, plasma, peripheral blood mononuclear cells (PBMCs) |
| Biomarkers | IL-1β, IL-18, NLRP3, ASC, caspase-1, GSDMD (all in CSF and plasma) |
| Correlation | UPDRS motor score, MoCA, DAT-SPECT imaging |
| Design | Randomized, double-blind, placebo-controlled |
| Primary Endpoint | Biomarker levels vs. clinical measures |
| Population | Early-stage PD (Hoehn & Yahr 1-2) with elevated inflammatory biomarkers |
| Intervention | MCC950 or placebo |
| Sample Size | 60 patients (30 per arm) |
| KG Connections | 2 knowledge graph edges |
| Databases | GeneCardsUniProtNCBI GeneHPASTRING |