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    Safety and tolerability of MRI-guided infusion of AAV2-hAADC into the mid-brain of nonhuman primate
    with Sebastian W. San, A. P. Kells, J. Bringas, L. Samaranch, P. Hadaczek, A. Ciesielska, M. J. Macayan, P. J. Pivirotto, J. Forsayeth, J. F. Wright, F. Green, G. Heller, and K. S. Bankiewicz
    Aromatic L-amino acid decarboxylase 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 in AADC-deficient children. Unfortunately, patients presented …Read more