A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys.

  title={A dose-ranging study of AAV-hAADC therapy in Parkinsonian monkeys.},
  author={John R. Forsayeth and J. L. Eberling and Laura M Sanftner and Zhu Zhen and Philip J Pivirotto and John R. Bringas and Janet Pagan Peter Higgs Stuart Cunningham and Krystof S. Bankiewicz},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  volume={14 4},
The main medication for idiopathic Parkinson disease is L-Dopa. Drug efficacy declines steadily in part because the converting enzyme, aromatic L-amino acid decarboxylase (AADC), is lost concomitant with substantia nigra atrophy. Over the past decade, we have developed a gene therapy approach in which AADC activity is restored to the brain by infusion into the striatum of a recombinant adeno-associated virus carrying human AADC cDNA. We report here the results of an investigation of the… CONTINUE READING


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