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Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency by MR-Guided Direct Delivery of AAV2-AADC to Midbrain Dopaminergic Neurons

2020-09-25

Abstract excerpt

<title>Abstract</title> <p><bold>Background</bold>: Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder characterized by deficient synthesis of dopamine and serotonin. It presents in early infancy, and causes severe developmental disability and lifelong motor, behavioral, and autonomic symptoms including oculogyric crises (OGC), sleep disorder, and mood disturbance.<bold>Objective</bo...

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Literature Corpus work: f8b89d70-1abf-5171-ac64-e3d05c62f7e2
DOI: 10.21203/rs.3.rs-73870/v1

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