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

(NIH/National Institute of Neurological Disorders and Stroke) 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.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news