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

被引：106

作者：

Kells, Adrian P. ^{[1
]}

Hadaczek, Piotr ^{[1
]}

Yin, Dali ^{[1
]}

Bringas, John ^{[1
]}

Varenika, Vanja ^{[1
]}

Forsayeth, John ^{[1
]}

Bankiewicz, Krystof S. ^{[1
]}

机构：

[1] Univ Calif San Francisco, Dept Neurol Surg, San Francisco, CA 94103 USA

来源：

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA | 2009年 / 106卷 / 07期

关键词：

adeno-associated viral vector; cortex; gene delivery; thalamocortical; CONVECTION-ENHANCED DELIVERY; ADENOASSOCIATED VIRUS VECTOR; FRONTAL-LOBE; PARKINSONS-DISEASE; AXONAL-TRANSPORT; MONKEY BRAIN; RAT-BRAIN; LIPOSOMES; TRIAL; TRANSDUCTION;

D O I：

10.1073/pnas.0810682106

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

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.

引用

页码：2407 / 2411

页数：5

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