Efficient gene therapy-based method for the delivery of therapeutics to primate cortex
Open Access
- 17 February 2009
- journal article
- research article
- Published by Proceedings of the National Academy of Sciences in Proceedings of the National Academy of Sciences of the United States of America
- Vol. 106 (7), 2407-2411
- https://doi.org/10.1073/pnas.0810682106
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.This publication has 41 references indexed in Scilit:
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