Summary Summary Short summary of a recent publication, written by scientific experts.
Gene therapy in children with L-amino acid decarboxylase (AADC) deficiency increases fractional anisotropy (FA) levels, which is linked to improved motor function despite lack of obvious structural changes.
AADC plays an integral role in dopamine and serotonin production, with deficiency resulting in severe motor dysfunction; findings validate a new technique that could serve of great benefit to children responding poorly to established therapies.
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