Gene therapy produces dramatic improvements in children with rare AADC deficiency

One child died seven months post-surgery, likely from underlying disease progression. Six of seven children experienced life-changing functional improvements including restored mobility, speech, and social interaction.

In the quiet architecture of the human brain, seven children carried a silence that was not chosen — their neurons intact, their will present, but the molecular key to movement missing entirely. At UCSF, researchers threaded a corrective gene into the precise chambers where dopamine is born, and in most of these children, the body began, slowly, to answer. The trial, published in Nature Communications in July 2021, stands as a reminder that some of medicine's most profound acts are not the conquest of disease, but the restoration of what was always meant to be there.

Gene therapy produces dramatic improvements in children with rare AADC deficiency

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