Safety and tolerability of putaminal AADC gene therapy for Parkinson disease.

@article{Christine2009SafetyAT,
  title={Safety and tolerability of putaminal AADC gene therapy for Parkinson disease.},
  author={Chadwick W. Christine and Philip A. Starr and Paul S. Larson and J. L. Eberling and William J. Jagust and Randall A. Hawkins and Henry F. Vanbrocklin and J. F. Wright and Krystof S. Bankiewicz and Michael J. Aminoff},
  journal={Neurology},
  year={2009},
  volume={73 20},
  pages={1662-9}
}
BACKGROUND In Parkinson disease (PD), the benefit of levodopa therapy becomes less marked over time, perhaps because degeneration of nigrostrial neurons causes progressive loss of aromatic l-amino acid decarboxylase (AADC), the enzyme that converts levodopa into dopamine. In a primate model of PD, intrastriatal infusion of an adeno-associated viral type 2 vector containing the human AADC gene (AAV-hAADC) results in robust response to low-dose levodopa without the side effects associated with… CONTINUE READING

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