title: Efficient gene therapy-based method for the delivery of therapeutics to primate cortex.
abstract: Transduction of the primate cortex with adeno-associated virus (AAV)-based gene therapy vectors has been challenging, because of the large size of the cortex. We report that a single infusion of AAV2 vector into thalamus results in widespread expression of transgene in the cortex through transduction of widely dispersed thalamocortical projections. This finding has important implications for the treatment of certain genetic and neurodegenerative diseases.
authors.affiliation: Department of Neurological Surgery, University of California, 1855 Folsom Street, San Francisco, CA 94103, USA.
authors.forename: Adrian P
authors.initials: AP
authors.lastname: Kells
authors.forename: Piotr
authors.initials: P
authors.lastname: Hadaczek
authors.forename: Dali
authors.initials: D
authors.lastname: Yin
authors.forename: John
authors.initials: J
authors.lastname: Bringas
authors.forename: Vanja
authors.initials: V
authors.lastname: Varenika
authors.forename: John
authors.initials: J
authors.lastname: Forsayeth
authors.forename: Krystof S
authors.initials: KS
authors.lastname: Bankiewicz
mesh_terms: D000818:Animals; D002540:Cerebral Cortex / Q000378:metabolism*; D000229:Dependovirus / Q000378:metabolism*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D051100:Glial Cell Line-Derived Neurotrophic Factor / Q000378:metabolism; D006801:Humans; D007150:Immunohistochemistry / Q000379:methods; D008253:Macaca mulatta; D008954:Models, Biological; D008957:Models, Genetic; D019636:Neurodegenerative Diseases / Q000378:metabolism / Q000628:therapy*; D009474:Neurons / Q000378:metabolism; D013788:Thalamus / Q000378:metabolism; D019076:Transgenes
chemical_list: D051100:Glial Cell Line-Derived Neurotrophic Factor