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The state of the art of adeno-associated virus-based vectors in gene therapy
The adeno-associated virus (AAV) has rapidly gained popularity in gene therapy since the establishment of the first AAV2 infectious clone, in 1982, due to some of their distinguishing characteristics such as lack of pathogenicity, wide range of infectivity, and ability to establish long-term transge...
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Formato: | Texto |
Lenguaje: | English |
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BioMed Central
2007
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2104528/ https://www.ncbi.nlm.nih.gov/pubmed/17939872 http://dx.doi.org/10.1186/1743-422X-4-99 |
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author | Coura, Renata dos Santos Nardi, Nance Beyer |
author_facet | Coura, Renata dos Santos Nardi, Nance Beyer |
author_sort | Coura, Renata dos Santos |
collection | PubMed |
description | The adeno-associated virus (AAV) has rapidly gained popularity in gene therapy since the establishment of the first AAV2 infectious clone, in 1982, due to some of their distinguishing characteristics such as lack of pathogenicity, wide range of infectivity, and ability to establish long-term transgene expression. Notably over the past decade, this virus has attracted considerable interest as a gene therapy vector, and about 85% of the currently available 2,041 PubMed references on adeno-associated viruses have been published during this time. The exponential progress of AAV-based vectors has been made possible by the advances in the knowledge of the virology and biology of this virus, which allows great improvement in AAV vectors construction and a better comprehension of their operation. Moreover, with the recent discovery of novel AAV serotypes, there is virtually one preferred serotype for nearly every organ or tissue to target. Thus, AAV-based vectors have been successfully overcoming the main gene therapy challenges such as transgene maintenance, safety and host immune response, and meeting the desirable vector system features of high level of safety combined with clinical efficacy and versatility in terms of potential applications. Consequently, AAV is increasingly becoming the vector of choice for a wide range of gene therapy approaches. This report will highlight the state of the art of AAV-based vectors studies and the advances on the use of AAV vectors for several gene therapy approaches. |
format | Text |
id | pubmed-2104528 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2007 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-21045282007-12-04 The state of the art of adeno-associated virus-based vectors in gene therapy Coura, Renata dos Santos Nardi, Nance Beyer Virol J Review The adeno-associated virus (AAV) has rapidly gained popularity in gene therapy since the establishment of the first AAV2 infectious clone, in 1982, due to some of their distinguishing characteristics such as lack of pathogenicity, wide range of infectivity, and ability to establish long-term transgene expression. Notably over the past decade, this virus has attracted considerable interest as a gene therapy vector, and about 85% of the currently available 2,041 PubMed references on adeno-associated viruses have been published during this time. The exponential progress of AAV-based vectors has been made possible by the advances in the knowledge of the virology and biology of this virus, which allows great improvement in AAV vectors construction and a better comprehension of their operation. Moreover, with the recent discovery of novel AAV serotypes, there is virtually one preferred serotype for nearly every organ or tissue to target. Thus, AAV-based vectors have been successfully overcoming the main gene therapy challenges such as transgene maintenance, safety and host immune response, and meeting the desirable vector system features of high level of safety combined with clinical efficacy and versatility in terms of potential applications. Consequently, AAV is increasingly becoming the vector of choice for a wide range of gene therapy approaches. This report will highlight the state of the art of AAV-based vectors studies and the advances on the use of AAV vectors for several gene therapy approaches. BioMed Central 2007-10-16 /pmc/articles/PMC2104528/ /pubmed/17939872 http://dx.doi.org/10.1186/1743-422X-4-99 Text en Copyright © 2007 Coura and Nardi; licensee BioMed Central Ltd. http://creativecommons.org/licenses/by/2.0 This is an Open Access article distributed under the terms of the Creative Commons Attribution License ( (http://creativecommons.org/licenses/by/2.0) ), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Review Coura, Renata dos Santos Nardi, Nance Beyer The state of the art of adeno-associated virus-based vectors in gene therapy |
title | The state of the art of adeno-associated virus-based vectors in gene therapy |
title_full | The state of the art of adeno-associated virus-based vectors in gene therapy |
title_fullStr | The state of the art of adeno-associated virus-based vectors in gene therapy |
title_full_unstemmed | The state of the art of adeno-associated virus-based vectors in gene therapy |
title_short | The state of the art of adeno-associated virus-based vectors in gene therapy |
title_sort | state of the art of adeno-associated virus-based vectors in gene therapy |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2104528/ https://www.ncbi.nlm.nih.gov/pubmed/17939872 http://dx.doi.org/10.1186/1743-422X-4-99 |
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