title: Gene therapy for neurodegenerative diseases.
abstract: 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.
authors.affiliation: Department of Neurosurgery, Emory University, 101 Woodruff Circle, Atlanta, GA 30322, USA.
authors.forename: Deirdre M
authors.initials: DM
authors.lastname: O'Connor
authors.affiliation: Department of Neurosurgery, Emory University, 101 Woodruff Circle, Atlanta, GA 30322, USA. Electronic address: nboulis@emory.edu.
authors.forename: Nicholas M
authors.initials: NM
authors.lastname: Boulis
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods; D006801:Humans; D019636:Neurodegenerative Diseases / Q000235:genetics* / Q000628:therapy*; D016896:Treatment Outcome
keywords: clinical trial; gene therapy; neurodegenerative disease