Parkinson’s is a disease that develops in part of the midbrain known as the substantia nigra when the cells producing dopamine start to die off. (Dopamine is a neurotransmitter that controls movement.) In the earlier stages of Parkinson’s, symptoms are more subtle and include difficulty making facial expressions, stiffening of the limbs and stooped posture. Symptoms are usually held in check by the drug levodopa. However, as the disease advances, symptoms worsen and levodopa becomes less effective. This is attributed to AADC – a primary enzyme that converts levodopa to dopamine. AADC declines as Parkinson’s runs its course.
Scientists at UC San Francisco have pioneered the use of gene therapy to address this issue. In phase I of this gene therapy trial, they deliver the gene for AADC using an inactive virus. Using a catheter, the gene is infused into a deep brain…
