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      Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

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          Abstract

          There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells, and has attracted a significant amount of attention in the field, especially in clinical-stage experimental therapeutic strategies. The ability to generate recombinant AAV particles lacking any viral genes and containing DNA sequences of interest for various therapeutic applications has thus far proven to be one of the safest strategies for gene therapies. This review will provide an overview of some important factors to consider in the use of AAV as a vector for gene therapy.

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          Clades of Adeno-associated viruses are widely disseminated in human tissues.

          Guangping Gao, Luk H Vandenberghe, Mauricio R Alvira … (2004)
          The potential for using Adeno-associated virus (AAV) as a vector for human gene therapy has stimulated interest in the Dependovirus genus. Serologic data suggest that AAV infections are prevalent in humans, although analyses of viruses and viral sequences from clinical samples are extremely limited. Molecular techniques were used in this study to successfully detect endogenous AAV sequences in 18% of all human tissues screened, with the liver and bone marrow being the most predominant sites. Sequence characterization of rescued AAV DNAs indicated a diverse array of molecular forms which segregate into clades whose members share functional and serologic similarities. One of the most predominant human clades is a hybrid of two previously described AAV serotypes, while another clade was found in humans and several species of nonhuman primates, suggesting a cross-species transmission of this virus. These data provide important information regarding the biology of parvoviruses in humans and their use as gene therapy vectors.
          Article 0 comments Cited 283 times – based on 0 reviews     Review now
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            Codon Bias as a Means to Fine-Tune Gene Expression.

            Tessa Quax, Nico J Claassens, Dieter Söll … (2015)
            The redundancy of the genetic code implies that most amino acids are encoded by multiple synonymous codons. In all domains of life, a biased frequency of synonymous codons is observed at the genome level, in functionally related genes (e.g., in operons), and within single genes. Other codon bias variants include biased codon pairs and codon co-occurrence. Although translation initiation is the key step in protein synthesis, it is generally accepted that codon bias contributes to translation efficiency by tuning the elongation rate of the process. Moreover, codon bias plays an important role in controlling a multitude of cellular processes, ranging from differential protein production to protein folding. Here we review currently known types of codon bias and how they may influence translation. We discuss how understanding the principles of codon bias and translation can contribute to improved protein production and developments in synthetic biology.
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              Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions.

              R. Samulski, C Summerford (1998)
              The human parvovirus adeno-associated virus (AAV) infects a broad range of cell types, including human, nonhuman primate, canine, murine, and avian. Although little is known about the initial events of virus infection, AAV is currently being developed as a vector for human gene therapy. Using defined mutant CHO cell lines and standard biochemical assays, we demonstrate that heparan sulfate proteoglycans mediate both AAV attachment to and infection of target cells. Competition experiments using heparin, a soluble receptor analog, demonstrated dose-dependent inhibition of AAV attachment and infection. Enzymatic removal of heparan but not chondroitin sulfate moieties from the cell surface greatly reduced AAV attachment and infectivity. Finally, mutant cell lines that do not produce heparan sulfate proteoglycans were significantly impaired for both AAV binding and infection. This is the first report that proteoglycan has a role in cellular attachment of a parvovirus. Together, these results demonstrate that membrane-associated heparan sulfate proteoglycan serves as the viral receptor for AAV type 2, and provide an explanation for the broad host range of AAV. Identification of heparan sulfate proteoglycan as a viral receptor should facilitate development of new reagents for virus purification and provide critical information on the use of AAV as a gene therapy vector.
              Article 0 comments Cited 181 times – based on 0 reviews     Review now
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                Author and article information

                Contributors
                Michael F. Naso:
                ORCID: http://orcid.org/0000-0001-9302-6186
                215-628-6952 , mnaso@its.jnj.com
                Journal
                Journal ID (nlm-ta): BioDrugs
                Journal ID (iso-abbrev): BioDrugs
                Title: Biodrugs
                Publisher: Springer International Publishing (Cham )
                ISSN (Print): 1173-8804
                ISSN (Electronic): 1179-190X
                Publication date (Electronic): 1 July 2017
                Publication date PMC-release: 1 July 2017
                Publication date (Print): 2017
                Volume: 31
                Issue: 4
                Pages: 317-334
                Affiliations
                [1 ]GRID grid.417429.d, , Janssen Research and Development, ; 200 McKean Road, Spring House, PA 19477 USA
                [2 ]BiStro Biotech Consulting, LLC, Bridgewater, NJ 08807 USA
                Author information
                http://orcid.org/0000-0001-9302-6186
                Article
                Publisher ID: 234
                DOI: 10.1007/s40259-017-0234-5
                PMC ID: 5548848
                PubMed ID: 28669112
                SO-VID: c422cd69-b0cd-44eb-9a4d-693ac08c6622
                Copyright © © The Author(s) 2017
                License:

                Open AccessThis article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (http://creativecommons.org/licenses/by-nc/4.0/), which permits any noncommercial use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.

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                Subject: Review Article
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                issue-copyright-statement © Springer International Publishing AG 2017

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