Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy
Abstract Three recent approvals and over 100 ongoing clinical trials make adeno‐associated virus (AAV)‐based vectors the leading gene delivery vehicles in gene therapy. Pharmaceutical companies are investing in this small and nonpathogenic gene shuttle to increase the therapeutic portfolios within t...
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Online Access: | https://doi.org/10.1002/advs.202004018 |
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doaj-3e9715ac034b476fb1e20d318f83b8c42021-05-05T07:56:41ZengWileyAdvanced Science2198-38442021-05-0189n/an/a10.1002/advs.202004018Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene TherapyHanna J. Wagner0Wilfried Weber1Martin Fussenegger2Department of Biosystems Science and Engineering ETH Zurich Mattenstrasse 26 Basel 4058 SwitzerlandFaculty of Biology University of Freiburg Schänzlestraße 1 Freiburg 79104 GermanyDepartment of Biosystems Science and Engineering ETH Zurich Mattenstrasse 26 Basel 4058 SwitzerlandAbstract Three recent approvals and over 100 ongoing clinical trials make adeno‐associated virus (AAV)‐based vectors the leading gene delivery vehicles in gene therapy. Pharmaceutical companies are investing in this small and nonpathogenic gene shuttle to increase the therapeutic portfolios within the coming years. This prospect of marking a new era in gene therapy has fostered both investigations of the fundamental AAV biology as well as engineering studies to enhance delivery vehicles. Driven by the high clinical potential, a new generation of synthetic‐biologically engineered AAV vectors is on the rise. Concepts from synthetic biology enable the control and fine‐tuning of vector function at different stages of cellular transduction and gene expression. It is anticipated that the emerging field of synthetic‐biologically engineered AAV vectors can shape future gene therapeutic approaches and thus the design of tomorrow's gene delivery vectors. This review describes and discusses the recent trends in capsid and vector genome engineering, with particular emphasis on synthetic‐biological approaches.https://doi.org/10.1002/advs.202004018AAV; adeno‐associated viruscapsid modificationsengineeringgene deliverymolecular switchesvector design |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Hanna J. Wagner Wilfried Weber Martin Fussenegger |
spellingShingle |
Hanna J. Wagner Wilfried Weber Martin Fussenegger Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy Advanced Science AAV; adeno‐associated virus capsid modifications engineering gene delivery molecular switches vector design |
author_facet |
Hanna J. Wagner Wilfried Weber Martin Fussenegger |
author_sort |
Hanna J. Wagner |
title |
Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy |
title_short |
Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy |
title_full |
Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy |
title_fullStr |
Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy |
title_full_unstemmed |
Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy |
title_sort |
synthetic biology: emerging concepts to design and advance adeno‐associated viral vectors for gene therapy |
publisher |
Wiley |
series |
Advanced Science |
issn |
2198-3844 |
publishDate |
2021-05-01 |
description |
Abstract Three recent approvals and over 100 ongoing clinical trials make adeno‐associated virus (AAV)‐based vectors the leading gene delivery vehicles in gene therapy. Pharmaceutical companies are investing in this small and nonpathogenic gene shuttle to increase the therapeutic portfolios within the coming years. This prospect of marking a new era in gene therapy has fostered both investigations of the fundamental AAV biology as well as engineering studies to enhance delivery vehicles. Driven by the high clinical potential, a new generation of synthetic‐biologically engineered AAV vectors is on the rise. Concepts from synthetic biology enable the control and fine‐tuning of vector function at different stages of cellular transduction and gene expression. It is anticipated that the emerging field of synthetic‐biologically engineered AAV vectors can shape future gene therapeutic approaches and thus the design of tomorrow's gene delivery vectors. This review describes and discusses the recent trends in capsid and vector genome engineering, with particular emphasis on synthetic‐biological approaches. |
topic |
AAV; adeno‐associated virus capsid modifications engineering gene delivery molecular switches vector design |
url |
https://doi.org/10.1002/advs.202004018 |
work_keys_str_mv |
AT hannajwagner syntheticbiologyemergingconceptstodesignandadvanceadenoassociatedviralvectorsforgenetherapy AT wilfriedweber syntheticbiologyemergingconceptstodesignandadvanceadenoassociatedviralvectorsforgenetherapy AT martinfussenegger syntheticbiologyemergingconceptstodesignandadvanceadenoassociatedviralvectorsforgenetherapy |
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