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New targeted gene therapy could reverse neurological diseases

A research team in the US have created a new, targeted method of gene therapy that could help children born with a rare genetic disorder called aromatic L-amino acid decarboxylase (AADC) deficiency. The disease is characterised by deficient synthesis of dopamine and serotonin and causes severe physical and developmental disabilities.

The study, conducted by researchers at The Ohio State University, US, involved the targeted delivery of gene therapy to the midbrain in seven children ages four to nine years-old who were infused with a viral vector. During the surgery, physicians infused a benign virus programmed with specific DNA into precisely targeted areas of the brain.

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