Gene therapy for L-amino acid decarboxylase deficiency in children

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.