Gene therapy for neurodegenerative diseases

• New therapeutic targets have recently been identified for neurodegenerative diseases.
• Gene therapy has benefited from advances in viral vectors and delivery methods.
• Current trials of AAV9-SMN represent a significant advance in SMA therapy.
• A tricistronic lentivirus for dopamine manipulation in Parkinsons (Prosavin) shows promise.

Gene therapy is, potentially, a powerful tool for treating neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), spinal muscular atrophy, Parkinson's disease (PD) and Alzheimer's disease (AD). To date, clinical trials have failed to show any improvement in outcome beyond the placebo effect. Efforts to improve outcomes are focusing on three main areas: vector design and the identification of new vector serotypes, mode of delivery of gene therapies, and identification of new therapeutic targets. These advances are being tested both individually and together to improve efficacy. These improvements may finally make gene therapy successful for these disorders.