Key result
Intraputamenal infusion of AAV2-hAADC in monkeys resulted in a dose-dependent increase in vector DNA, with 99% detected in the target site and basal ganglia tissues at 6 months.
Why the study?
Does intraputamenal infusion of AAV2-hAADC via convection-enhanced delivery result in targeted brain biodistribution in monkeys with parkinsonian syndrome?
Does intraputamenal infusion of AAV2-hAADC via convection-enhanced delivery result in targeted brain biodistribution in monkeys with parkinsonian syndrome?
Convection-enhanced delivery of AAV2-hAADC achieves highly targeted brain biodistribution in parkinsonian monkeys, supporting its progression to Phase I clinical trials.
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Supports dose selection for human AAV2-hAADC trials in late-stage Parkinson's; extends primate biodistribution data but leaves clinical translation open.
Cunningham et al. (2008) studied Parkinsonian syndrome (n=12). AAV2-hAADC via intraputamenal infusion vs. AAV2-GFP was evaluated on Biodistribution of AAV2-hAADC (vector DNA via Q-PCR). Intraputamenal infusion of AAV2-hAADC in monkeys resulted in a dose-dependent increase in vector DNA, with 99% detected in the target site and basal ganglia tissues at 6 months.
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