Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

Gene therapy has the potential to provide therapeutic benefit to millions of people with
neurodegenerative diseases through several means, including direct correction of …

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder
characterized by deficient synthesis of dopamine and serotonin. It presents in early infancy …

The current mainstay treatment of Parkinson's disease (PD) consists of dopamine
replacement therapy which, in addition to causing several side effects, does not delay …

The growing research interest in nanomedicine for the treatment of cancer and inflammatory-
related pathologies is yielding encouraging results. Unfortunately, enthusiasm is tempered …

Parkinson's disease (PD) is a neurodegenerative illness and has a common onset between
the ages of 55 and 65 years. There is progressive development of both motor and non-motor …

Background: In Parkinson disease (PD), the benefit of levodopa therapy becomes less
marked over time, perhaps because degeneration of nigrostrial neurons causes progressive …

We report the results of a long-term follow-up of subjects in a phase 1 study of AAV2-hAADC
(adeno-associated virus type 2–human aromatic l-amino acid decarboxylase) gene therapy …

Gene transfer of dopamine-synthesizing enzymes into the striatal neurons has led to
behavioral recovery in animal models of Parkinson's disease (PD). We evaluated the safety …

Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …