AAV5-GCH1-TH

**AAV5-GCH1-TH** is an experimental, neuron-targeted adeno-associated virus serotype 5 gene therapy for **Parkinson's disease**. It is administered by bilateral stereotactic injection into the putamen and carries separate human **GCH1** and **TH** expression cassettes driven by synapsin-1 promoters. Transduced striatal neurons express GTP cyclohydrolase 1 and tyrosine hydroxylase to generate tetrahydrobiopterin and continuously synthesize L-DOPA locally; endogenous aromatic L-amino acid decarboxylase is then required to convert the vector-derived L-DOPA to dopamine. In rodent and MPTP-treated rhesus macaque models, the vector produced dose-dependent motor improvement without worsening pre-existing L-DOPA-induced dyskinesia. ([pmc.ncbi.nlm.nih.gov](https://pmc.ncbi.nlm.nih.gov/articles/PMC6658866/))

Other names

AAV-GCH1-THAAV-GCH-1-THAAV-GCH 1-THAAV5 Syn1-GCH1|Syn1-TH-WAAV5-GCH1-THAAV-5-GCH1-THAAV 5-GCH1-THrAAV-TH/GCH1Syn1-GCH1|Syn1-TH-W