Takeaway
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.
Why this matters ?
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.