SHUTTERSTOCK, IDESIGN
An inherited disorder known as aromatic L-amino acid decarboxylase deficiency, or AADC, leaves patients unable to produce the neurotransmitter dopamine, leaving them nearly paralyzed until they die in early childhood. But a new gene therapy, which involves the delivery of the missing L-amino acid decarboxylase enzyme that converts the chemical precursor L-DOPA to dopamine, helped four Taiwanese children, aged 4 to 6 years, move their heads and sit up on their own, according to a study published today (May 16) in Science Translational Medicine.
“The children in this study have the most severe form of inherited movement disorder known, and the only treatments so far have been supportive ones,” pediatric cardiologist Barry Byrne, director of the University of Florida’s Powell Gene Therapy Center, said in ...
