STP-001 + clozapine

STP-001 + clozapine is an experimental chemogenetic combination therapy developed by Ruijin Hospital for the treatment of Parkinson's disease. The therapy utilizes a two-step approach: first, an adeno-associated virus (AAV) vector, designated STP-001, is stereotactically injected into the bilateral subthalamic nucleus (STN). This vector carries the human synapsin (hSyn) promoter and the inhibitory chemogenetic effector hM4Di, a Designer Receptor Exclusively Activated by Designer Drugs (DREADD). Once expressed in the STN neurons, the hM4Di receptor remains inactive until the second component, a very low dose of oral clozapine, is administered. Clozapine acts as a specific ligand that activates the hM4Di receptors, triggering Gi-protein-mediated signaling to suppress the pathological neuronal overactivity of the STN characteristic of Parkinson's disease. This strategy aims to provide precise, reversible, and adjustable control over motor symptoms while avoiding the side effects associated with high-dose clozapine or the invasiveness of permanent deep brain stimulation hardware.

Other names

STP-001 ChemogeneticsSTP001 ChemogeneticsSTP 001 ChemogeneticsAAV-hSyn-hM4Di gene therapyAAV-hSyn-hM-4Di gene therapyAAV-hSyn-hM 4Di gene therapy