title: Parkinson's disease: gene therapies.
abstract: With the recent development of effective gene delivery systems, gene therapy for the central nervous system is finding novel applications. Here, we review existing viral vectors and discuss gene therapy strategies that have been proposed for Parkinson's disease. To date, most of the clinical trials were based on viral vectors to deliver therapeutic transgenes to neurons within the basal ganglia. Initial trials used genes to relieve the major motor symptoms caused by nigrostriatal degeneration. Although these new genetic approaches still need to prove more effective than existing symptomatic treatments, there is a need for disease-modifying strategies. The investigation of the genetic factors implicated in Parkinson's disease is providing precious insights in disease pathology that, combined with innovative gene delivery systems, will hopefully offer novel opportunities for gene therapy interventions to slow down, or even halt disease progression.
authors.affiliation: Neurodegenerative Studies Laboratory, Brain Mind Institute, Ecole Polytechnique Fédérale de Lausanne (EPFL), CH-1015 Lausanne, Switzerland.
authors.forename: Philippe G
authors.initials: PG
authors.lastname: Coune
authors.forename: Bernard L
authors.initials: BL
authors.lastname: Schneider
authors.forename: Patrick
authors.initials: P
authors.lastname: Aebischer
mesh_terms: D000229:Dependovirus; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000627:therapeutic use*; D051100:Glial Cell Line-Derived Neurotrophic Factor / Q000235:genetics / Q000627:therapeutic use; D005968:Glutamate Decarboxylase / Q000235:genetics / Q000378:metabolism; D006801:Humans; D016086:Lentivirus; D007980:Levodopa / Q000235:genetics / Q000378:metabolism; D010300:Parkinson Disease / Q000235:genetics* / Q000628:therapy*; D044767:Ubiquitin-Protein Ligases / Q000235:genetics; D051844:alpha-Synuclein / Q000235:genetics
chemical_list: D051100:Glial Cell Line-Derived Neurotrophic Factor; D051844:alpha-Synuclein; D007980:Levodopa; D044767:Ubiquitin-Protein Ligases; C111567:parkin protein; D005968:Glutamate Decarboxylase