Abstract
Adeno-associated virus (AAV) vectors are promising human gene delivery vehicles due to their ability to establish long-term gene expression in a wide variety of target tissues; however, the broad native viral tropism raises concerns over the feasibility and safety of their systemic administration. To overcome this issue, much effort has been made to redirect AAVs toward specific tissues. This review presents several design strategies that have been applied to generate AAVs that target specific tissues and cells while inhibiting the transduction of non-target tissues. Multiple methods of vector capsid engineering have shown promise in vitro, including indirect targeting by adaptor systems and direct targeting by the insertion of antibodies or receptor-specific small peptide motifs. Other strategies, including creating mosaic or chimeric capsids and directed evolution, have also been used to successfully retarget AAV vectors. This research will further expand the clinical applications of AAV vectors by enhancing the control over tissue-specific gene delivery.
Keywords: Adeno-assocaited virus, capsid, human gene delivery, serotype, targeting, tropism.
Current Pharmaceutical Design
Title:Generation of Targeted Adeno-Associated Virus (AAV) Vectors for Human Gene Therapy
Volume: 21 Issue: 22
Author(s): Yarong Liu, Natnaree Siriwon, Jennifer A. Rohrs and Pin Wang
Affiliation:
Keywords: Adeno-assocaited virus, capsid, human gene delivery, serotype, targeting, tropism.
Abstract: Adeno-associated virus (AAV) vectors are promising human gene delivery vehicles due to their ability to establish long-term gene expression in a wide variety of target tissues; however, the broad native viral tropism raises concerns over the feasibility and safety of their systemic administration. To overcome this issue, much effort has been made to redirect AAVs toward specific tissues. This review presents several design strategies that have been applied to generate AAVs that target specific tissues and cells while inhibiting the transduction of non-target tissues. Multiple methods of vector capsid engineering have shown promise in vitro, including indirect targeting by adaptor systems and direct targeting by the insertion of antibodies or receptor-specific small peptide motifs. Other strategies, including creating mosaic or chimeric capsids and directed evolution, have also been used to successfully retarget AAV vectors. This research will further expand the clinical applications of AAV vectors by enhancing the control over tissue-specific gene delivery.
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Cite this article as:
Liu Yarong, Siriwon Natnaree, Rohrs A. Jennifer and Wang Pin, Generation of Targeted Adeno-Associated Virus (AAV) Vectors for Human Gene Therapy, Current Pharmaceutical Design 2015; 21 (22) . https://dx.doi.org/10.2174/1381612821666150531171653
DOI https://dx.doi.org/10.2174/1381612821666150531171653 |
Print ISSN 1381-6128 |
Publisher Name Bentham Science Publisher |
Online ISSN 1873-4286 |
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