According to a new study published in Nature Communications, injecting the DDC gene into the brain could be a safe and efficient method to treat aromatic L-amino acid decarboxylase (AADC) deficiency. Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare autosomal recessive neurodevelopmental disorder characterized by low levels of dopamine, epinephrine, norepinephrine, and serotonin. Children with this disorder exhibit severe developmental delays, psychological disorders, and motor disabilities such as sleep disruptions, speech, and walking difficulties.
In this study, seven children received an infusion of the DDC gene packaged in an adenovirus for delivery into the brain cells. The DDC gene is incorporated into the DNA of cells and instructs them to generate AADC, an enzyme required to produce serotonin and dopamine. The researchers employed real-time MR imaging in…
