Dopamine metabolism & DOPAL Gene therapy
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Lentiviral delivery of a three-enzyme dopamine biosynthesis cassette (TH, GCH1, AADC) to the putamen enables endogenous, continuous dopamine production in non-dopaminergic striatal neurons, providing a cell-autonomous replacement for the lost nigrostriatal input.
OXB-102 uses an EIAV-derived lentiviral vector to transduce putaminal neurons with TH, GCH1, and AADC, equipping them to convert endogenous tyrosine into dopamine without dependence on the surviving substantia nigra projection neurons that progressively degenerate in Parkinson's disease.