Switching focus in the brain, researchers herald a possible gene therapy breakthrough in treating an ultra-rare disease
A group of investigators testing a gene therapy for ultra-rare cases of aromatic amino acid decarboxylase deficiency has seen some remarkable results in their first group of 7 young patients.
According to the study published in Nature on Monday, the researchers decided to take a novel approach to tackle the disease, which so far has resisted just about everything tried against it. The disease is characterized by a deficiency in AADC, an enzyme that “catalyzes the synthesis of dopamine and serotonin from their respective precursors.”
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