Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial.

@article{Kaplitt2007SafetyAT,
  title={Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial.},
  author={Michael G. Kaplitt and Andrew S Feigin and Chengke Tang and Helen L Fitzsimons and Paul J. Mattis and Patricia A. Lawlor and Ross J Bland and Deborah Young and Kristin Strybing and David Eidelberg and Matthew John During},
  journal={Lancet},
  year={2007},
  volume={369 9579},
  pages={2097-105}
}
BACKGROUND Dopaminergic neuronal loss in Parkinson's disease leads to changes in the circuitry of the basal ganglia, such as decreased inhibitory GABAergic input to the subthalamic nucleus. We aimed to measure the safety, tolerability, and potential efficacy of transfer of glutamic acid decarboxylase (GAD) gene with adeno-associated virus (AAV) into the subthalamic nucleus of patients with Parkinson's disease. METHODS We did an open label, safety and tolerability trial of unilateral… CONTINUE READING
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