title: Application of ex vivo gene therapy in the treatment of Parkinson's disease.
abstract: Ex vivo gene therapy approaches hold great promise for the treatment of neurodegenerative diseases where there is currently no cure or adequate treatment for affected individuals. In this review we have focused on the use of ex vivo gene transfer techniques in Parkinson's disease models; however, the issues and approaches outlined are applicable to other neurodegenerative disorders. In utilizing the ex vivo strategy two considerations are critical for delivery of therapeutic levels of transgene product to the target: (i) the vector system and (ii) the cell type for grafting. We describe herein different vector systems that are currently available and briefly review the various cell types that have been transduced and grafted into the striatum of animals with experimental Parkinson's disease. The strategies for application of gene therapy techniques to a treatment for Parkinson's disease have expanded beyond the classical dopamine replacement toward the use of neurotrophic factors in enhancing cell function or preventing cell death. In addition, we explore the utility of CNS-derived neural progenitors as alternative cell types for ex vivo gene therapy in an animal model of Parkinson's disease.
authors.affiliation: Salk Institute for Biological Studies, Laboratory of Genetics, San Diego, California 92186-5800, USA.
authors.forename: H K
authors.initials: HK
authors.lastname: Raymon
authors.forename: S
authors.initials: S
authors.lastname: Thode
authors.forename: F H
authors.initials: FH
authors.lastname: Gage
mesh_terms: D000818:Animals; D003342:Corpus Striatum / Q000378:metabolism; D004298:Dopamine / Q000378:metabolism; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D009414:Nerve Growth Factors / Q000235:genetics; D009474:Neurons / Q000637:transplantation; D010300:Parkinson Disease / Q000601:surgery / Q000628:therapy*; D033581:Stem Cell Transplantation
chemical_list: D009414:Nerve Growth Factors; D004298:Dopamine