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Localized Intra-Arterial Gene Delivery Using AAV.

作者信息

Hosaka Koji, Manfredsson Fredric P, Hoh Brian L

机构信息

Department of Neurosurgery, College of Medicine, University of Florida, Gainesville, FL, USA.

Department of Translational Science and Molecular Medicine, College of Human Medicine, Michigan State University, East Lansing, MI, USA.

出版信息

Methods Mol Biol. 2019;1937:259-265. doi: 10.1007/978-1-4939-9065-8_16.

DOI:10.1007/978-1-4939-9065-8_16
PMID:30706402
Abstract

In vivo gene therapy is a tremendous tool for a wide variety of genetic modifications. However, often a specific and precise local administration of the viral vector is necessary to deliver the genetic payload in vivo. For many animal studies using viral vectors, such as those investigating neurological disorders, the vector is targeted directly into the tissue/organ of interest. On the other hand, in vascular disease research, viral vectors are administered systemically, either via a tail vein injection or through catheter-mediated infusion, which results in off-target transduction of cells and tissues. Targeting cells in the vascular wall without off-target activity, however, requires localized delivery in order to efficiently target cells of the internal vasculature. Here we describe a novel murine in vivo targeted intra-arterial viral vector delivery method, which has been developed in order to be able to perform more intricate studies in cardiovascular disease.

摘要

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