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Gene therapy tackles 2 rare childhood diseases 2 different ways – The Science Board

Two new studies point to the efficient use of gene therapy to treat deadly childhood genetic diseases. The first study used direct…

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Gene therapy No. 1: Direct DDC gene therapy via viral vector
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare neurodevelopmental disorder characterized by the deficient synthesis of dopamine to serotonin. The deficiency impacts only 135 children…

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