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AAV-SYN-hM4Di
- Development stage
- Unknown
- Lead developer
- Xuanwu Hospital
- Modality
- Gene Therapies
- Administration
- Intralesional
01
Overview
AAV-SYN-hM4Di is an investigational adeno-associated virus (AAV) vector-based gene therapy developed for the treatment of refractory temporal lobe epilepsy (TLE). The construct utilizes a human synapsin (hSyn) promoter to drive the neuron-specific expression of hM4Di, a Designer Receptor Exclusively Activated by Designer Drugs (DREADD). hM4Di is a modified human muscarinic M4 receptor engineered to lose its affinity for endogenous acetylcholine while gaining high sensitivity to otherwise pharmacologically inert ligands, such as clozapine or clozapine N-oxide (CNO). When the ligand is administered, it activates the hM4Di receptor, which couples to the Gi signaling pathway to induce neuronal hyperpolarization and inhibit synaptic release. This chemogenetic approach allows for the selective and reversible inhibition of overactive epileptogenic neurons in the hippocampus. In clinical settings, the vector is delivered via robot-assisted convection-enhanced delivery (CED) directly into the affected brain region.
- Other names
- AAV-hSyn-hM4DiAAV-hSyn-hM-4DiAAV-hSyn-hM 4DiAAV-hSyn-hM4D(Gi)
02
Targets
hM4Di (Human muscarinic acetylcholine receptor M4 designer receptor exclusively activated by designer drugs)
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