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Gene Therapy Restores Dopamine Levels in Kids With Rare Disorder – Technology Networks

A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe…

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This study describes the findings from the targeted delivery of gene therapy to midbrain to treat a rare deadly neurodevelopmental disorder in children with a neurogenetic disease, aromatic L-amino acid decarboxylase (AADC) deficiency characterized by deficient…

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