AAV5 Syn1-GCH1 + Syn1-TH-W

**AAV5 Syn1-GCH1 + Syn1-TH-W** is an experimental **AAV5-based in vivo gene therapy** for **Parkinson's disease** designed to provide continuous local **l-DOPA** production within the putamen after a one-time stereotactic intraputaminal administration. The vector carries two human transgenes in a single AAV5 construct: **GCH1** driven by the **human synapsin 1 promoter** and **TH** driven by a second **human synapsin 1 promoter**, with the TH cassette including **WPRE**. By expressing **guanosine triphosphate cyclohydrolase 1** and **tyrosine hydroxylase** in striatal neurons, the therapy is intended to increase local synthesis of l-DOPA, which can then be converted by endogenous aromatic L-amino acid decarboxylase to dopamine in the dopamine-depleted striatum. In the cited 2019 nonhuman primate study, the construct showed dose-dependent improvement of parkinsonian motor deficits without worsening pre-existing l-DOPA-induced dyskinesia, and the work was funded by **Genepod Therapeutics**. The program appears to be preclinical/proof-of-concept rather than an approved or branded clinical product.

Other names

AAV5 Syn1-GCH1|Syn1-TH-WAAV5 GCH1-TH vectorAAV-5 GCH1-TH vectorAAV 5 GCH1-TH vectorAAV5 TH/GCH1 vectorAAV5 l-DOPA-producing vectorAAV-5 l-DOPA-producing vectorAAV 5 l-DOPA-producing vectorAAV5 continuous l-DOPA delivery vectorAAV-5 continuous l-DOPA delivery vectorAAV 5 continuous l-DOPA delivery vector