抄録
Dopamine, the major neurotransmitter depleted in Parkinson's disease, can be synthesized and regulated in vivo with a combination of intra-striatal AAV2-hAADC gene therapy and administration of the dopamine precursor L-dopa. When tested in MPTP-lesioned monkeys, this approach resulted in long-term improvement in clinical rating scores, significantly lowered L-dopa requirements, and a reduction of L-dopa-induced side effects. Positron emission tomography with [^<18>F]-FMT confirmed persistent AADC activity, demonstrating for the first time that infusion of AAV2 vector into primate brain results in at least 6 years of transgene expression. Armed with these data, a Phase 1 open-label does-escalation trial is now underway and interim results are expected in the near future.
