Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate

@article{Sebastin2014SafetyAT,
  title={Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate},
  author={Waldy San Sebasti{\'a}n and Adrian Philip Kells and John R. Bringas and Lluis Samaranch and Piotr Hadaczek and Agnieszka Ciesielska and Michael J Macayan and Phillip J Pivirotto and John R. Forsayeth and Sheryl Osborne and J. Fraser Wright and Foad Green and Gregory Heller and Krystof S. Bankiewicz},
  journal={Molecular Therapy. Methods \& Clinical Development},
  year={2014},
  volume={3}
}
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, autosomal-recessive neurological disorder caused by mutations in the DDC gene that leads to an inability to synthesize catecholamines and serotonin. As a result, patients suffer compromised development, particularly in motor function. A recent gene replacement clinical trial explored putaminal delivery of recombinant adeno-associated virus serotype 2 vector encoding human AADC (AAV2-hAADC) in AADC-deficient children. Unfortunately… 

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