NIH-funded study finds gene therapy may restore missing enzyme in rare disease ...Middle East

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NIH-funded study finds gene therapy may restore missing enzyme in rare disease
A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency. AADC deficiency is a rare neurological disorder that develops in infancy and leads to near absent levels of certain brain chemicals, serotonin and dopamine, that are critical for movement, behavior, and sleep.

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