AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

@article{Lewitt2011AAV2GADGT,
  title={AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial},
  author={Peter A. Lewitt and Ali R. Rezai and Maureen A. Leehey and Steven G. Ojemann and Andrew S. Feigin and S. Tatter},
  journal={The Lancet Neurology},
  year={2011},
  volume={10},
  pages={309-319}
}
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Findings show that clinical benefits after gene therapy with STN AAV2-GAD in PD patients persist at 12 months, with baseline metabolism in the prefrontal cortex correlated with changes in motor UPDRS scores; the higher the baseline PFC metabolism, the better the clinical outcome.
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The efficacy outcomes are encouraging and indicate that the AAV vector-mediated gene transfer of AADC may benefit advanced PD patients, and a similar approach, delivering A AV vector carrying A ADC gene into the putamen ameliorated the symptoms in children with AADc deficiency.
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Analysis of all FDG PET images over 12 months revealed significant metabolic declines in the thalamus, striatum, and prefrontal, anterior cingulate, and orbitofrontal cortices in the AAV2-GAD group compared with the sham group.
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TLDR
Parkinson's disease gene therapy has moved from purely animal research three decades ago, to initial human studies two decades ago to many applications moving into late stage trials, currently and recent successes and promising new technology should only accelerate the advance of gene therapy into active clinical practice.
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