alexa Improving AAV Gene Therapy: Graduating From Transgene Expression “Everywhere, All The Time” To “Disease-Specific” | OMICS International | Abstract
ISSN: 2168-9849

Cloning & Transgenesis
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Editorial

Improving AAV Gene Therapy: Graduating From Transgene Expression “Everywhere, All The Time” To “Disease-Specific”

Hermonat PL1,2,3*, Straub KD1,2 and Chiriva-Internati M4,5
1Central Arkansas Veterans Healthcare System, Little Rock, AR, USA
2Department of Internal Medicine, University of Arkansas for Medical Sciences, USA
3Department of Obstetrics and Gynecology, University of Arkansas for Medical Sciences, USA
4Department of Internal Medicine, Texas Tech University, Lubbock, TX, USA
5Kiromic, LLC, Lubbock, TX, USA
Corresponding Author : Hermonat PL
Department of Internal Medicine
University of Arkansas for Medical Sciences, USA
Tel: +501-686-5046
E-mail: [email protected]
Received September 25, 2014; Accepted September 29, 2014; Published October 1, 2014
Citation: Hermonat PL (2014) Improving AAV Gene Therapy: Graduating From Transgene Expression “Everywhere, All The Time” To “Disease-Specific”. Clon Transgen 3:e114. doi:10.4172/2168-9849.1000e114
Copyright: © 2014 Hermonat PL, et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.

Abstract

Adeno-associated virus (AAV) is now one of the top gene delivery/ gene therapy vectors in use today [1-6]. The hallmark goal of gene therapy is to deliver a therapeutic gene which then counteracts a negative phenotype or disease within the patient or animal model. While there are many types of diseases to treat, each disease is somewhat different and there are a variety of delivery/expression strategies which can be undertaken. AAV capsid type and tropism clearly play a valuable role in gene therapy; however the strategy for expressing the transgene is even more important. Here we discuss the three most prominent types of gene expression approaches, that is, which transcriptional promoter should be chosen for expressing the therapeutic gene. The issue is that many, perhaps all, therapeutic genes will likely have consequences, adverse reactions, if expressed at high levels. Yet the gene therapy agents must be safe, and not induce widespread unintended damage.

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