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Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors
In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential gene therapy for a number of human diseases. To date, the safety of AAV vectors in 176 phase I, II, and III clinical tr...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2019
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6378346/ https://www.ncbi.nlm.nih.gov/pubmed/30815511 http://dx.doi.org/10.1016/j.omtm.2019.01.008 |
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author | Büning, Hildegard Srivastava, Arun |
author_facet | Büning, Hildegard Srivastava, Arun |
author_sort | Büning, Hildegard |
collection | PubMed |
description | In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential gene therapy for a number of human diseases. To date, the safety of AAV vectors in 176 phase I, II, and III clinical trials and their efficacy in at least eight human diseases are now firmly documented. Despite these remarkable achievements, it has also become abundantly clear that the full potential of first generation AAV vectors composed of naturally occurring capsids is not likely to be realized, since the wild-type AAV did not evolve for the purpose of therapeutic gene delivery. In this article, we provide a brief historical account of the progress that has been made in the development of capsid-modified, next-generation AAV vectors to ensure both the safety and efficacy of these vectors in targeting a wide variety of human diseases. |
format | Online Article Text |
id | pubmed-6378346 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | American Society of Gene & Cell Therapy |
record_format | MEDLINE/PubMed |
spelling | pubmed-63783462019-02-27 Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors Büning, Hildegard Srivastava, Arun Mol Ther Methods Clin Dev Article In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential gene therapy for a number of human diseases. To date, the safety of AAV vectors in 176 phase I, II, and III clinical trials and their efficacy in at least eight human diseases are now firmly documented. Despite these remarkable achievements, it has also become abundantly clear that the full potential of first generation AAV vectors composed of naturally occurring capsids is not likely to be realized, since the wild-type AAV did not evolve for the purpose of therapeutic gene delivery. In this article, we provide a brief historical account of the progress that has been made in the development of capsid-modified, next-generation AAV vectors to ensure both the safety and efficacy of these vectors in targeting a wide variety of human diseases. American Society of Gene & Cell Therapy 2019-01-26 /pmc/articles/PMC6378346/ /pubmed/30815511 http://dx.doi.org/10.1016/j.omtm.2019.01.008 Text en © 2019 The Authors http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/). |
spellingShingle | Article Büning, Hildegard Srivastava, Arun Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors |
title | Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors |
title_full | Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors |
title_fullStr | Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors |
title_full_unstemmed | Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors |
title_short | Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors |
title_sort | capsid modifications for targeting and improving the efficacy of aav vectors |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6378346/ https://www.ncbi.nlm.nih.gov/pubmed/30815511 http://dx.doi.org/10.1016/j.omtm.2019.01.008 |
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