I posted this as a reply to Bass of Spade's optimistic post regarding gene therapy, but I think it's worthy of being the subject of a separate post.
Gene Therapy Offers Long-Awaited Hope for Children with Rare, Incurable Disorder. Patients with Parkinson’s, Alzheimer’s May Benefit from Novel Treatment.
"The trial borrowed from gene delivery techniques used to treat Parkinson’s disease, pioneered by senior author Krystof Bankiewicz, MD, PhD, of the UCSF Department of Neurological Surgery and the Weill Institute for Neurosciences, and of the Department of Neurological Surgery at Ohio State University. Both conditions are associated with deficiencies of AADC, which converts levodopa into dopamine, a neurotransmitter involved in movement, mood, learning and concentration."