• DOI:10.1038/mtm.2014.49
Corpus ID: 7662721

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

@article{Sebastian2014SafetyAT,
  title={Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate},
  author={Waldy San Sebastian and A. Kells and J. Bringas and L. Samaranch and P. Hadaczek and A. Ciesielska and M. Macayan and P. Pivirotto and J. Forsayeth and Sheryl Osborne and J. F. Wright and F. Green and G. Heller and K. Bankiewicz},
  journal={Molecular Therapy. Methods & Clinical Development},
  year={2014},
  volume={3}
}

Waldy San Sebastian, A. Kells, +11 authors K. Bankiewicz

Published 2014

Medicine

Molecular Therapy. Methods & Clinical Development

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… CONTINUE READING

View on PubMed

doi.org

Save to Library

Create Alert

Cite

Launch Research Feed