Efficient gene therapy-based method for the delivery of therapeutics to primate cortex.
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.
Kells
Adrian P
AP
Department of Neurological Surgery, University of California, 1855 Folsom Street, San Francisco, CA 94103, USA.
Hadaczek
Piotr
P
Yin
Dali
D
Bringas
John
J
Varenika
Vanja
V
Forsayeth
John
J
Bankiewicz
Krystof S
KS
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
D051100:Glial Cell Line-Derived Neurotrophic Factor