AAV9-hEPO

AAV9-hEPO is an investigational **gene therapy vector** composed of an **adeno-associated virus serotype 9 (AAV9)** backbone encoding the gene for **human erythropoietin (hEPO)**. This vector is designed to induce expression of human erythropoietin in target tissues, leading to increased erythropoietin production. **Mechanism of Action:** Following delivery, the AAV9 vector efficiently transduces neurons and other cell types, introducing the hEPO gene, which promotes erythropoietin synthesis. Preclinical studies have demonstrated neuroprotective effects in a rat model of Parkinson’s disease via protection of dopaminergic neurons in the substantia nigra. The therapeutic effect is attributed to hEPO's roles in neuroprotection, anti-apoptosis, and anti-inflammatory responses. The platform is experimental and has not been approved for clinical use.

Other names

AAV9-hEPOAAV-9-hEPOAAV 9-hEPO