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Midbrain Gene Therapy Improved Symptoms, Motor Function in Rare Genetic Disease

Medscape News
 2021-07-21

NEW YORK (Reuters Health) - Gene therapy delivered to the midbrain was safe and led to symptom and motor function improvement in children with a rare genetic disorder characterized by deficiencies in dopamine and serotonin synthesis. The disorder, aromatic L-amino acid decarboxylase (AADC) deficiency, presents in early infancy and causes...

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