Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

基于基因治疗的有效方法,用于将治疗剂输送到灵长类动物皮层

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作者:Adrian P Kells, Piotr Hadaczek, Dali Yin, John Bringas, Vanja Varenika, John Forsayeth, Krystof S Bankiewicz

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.

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