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Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery

Recombinant adeno-associated virus (rAAV) is an attractive tool for basic science and translational medicine including gene therapy, due to the versatility in its cell and organ transduction. Previous work indicates that rAAV transduction patterns are highly dependent on route of administration. Bas...

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Detalles Bibliográficos
Autores principales: Ai, Jianzhong, Li, Jia, Gessler, Dominic J., Su, Qin, Wei, Qiang, Li, Hong, Gao, Guangping
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5220346/
https://www.ncbi.nlm.nih.gov/pubmed/28067312
http://dx.doi.org/10.1038/srep40336
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author Ai, Jianzhong
Li, Jia
Gessler, Dominic J.
Su, Qin
Wei, Qiang
Li, Hong
Gao, Guangping
author_facet Ai, Jianzhong
Li, Jia
Gessler, Dominic J.
Su, Qin
Wei, Qiang
Li, Hong
Gao, Guangping
author_sort Ai, Jianzhong
collection PubMed
description Recombinant adeno-associated virus (rAAV) is an attractive tool for basic science and translational medicine including gene therapy, due to the versatility in its cell and organ transduction. Previous work indicates that rAAV transduction patterns are highly dependent on route of administration. Based on this relationship, we hypothesized that intraperitoneal (IP) administration of rAAV produces unique patterns of tissue tropism. To test this hypothesis, we investigated the transduction efficiency of 12 rAAV serotypes carrying an enhanced green fluorescent protein (EGFP) reporter gene in a panel of 12 organs after IP injection. Our data suggest that IP administration emphasizes transduction patterns that are different from previously reported intravascular delivery methods. Using this approach, rAAV efficiently transduces the liver, pancreas, skeletal muscle, heart and diaphragm without causing significant histopathological changes. Of note, rAAVrh.10 showed excellent muscle transduction following IP administration, highlighting its potential as a new muscle-targeting vector.
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spelling pubmed-52203462017-01-11 Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery Ai, Jianzhong Li, Jia Gessler, Dominic J. Su, Qin Wei, Qiang Li, Hong Gao, Guangping Sci Rep Article Recombinant adeno-associated virus (rAAV) is an attractive tool for basic science and translational medicine including gene therapy, due to the versatility in its cell and organ transduction. Previous work indicates that rAAV transduction patterns are highly dependent on route of administration. Based on this relationship, we hypothesized that intraperitoneal (IP) administration of rAAV produces unique patterns of tissue tropism. To test this hypothesis, we investigated the transduction efficiency of 12 rAAV serotypes carrying an enhanced green fluorescent protein (EGFP) reporter gene in a panel of 12 organs after IP injection. Our data suggest that IP administration emphasizes transduction patterns that are different from previously reported intravascular delivery methods. Using this approach, rAAV efficiently transduces the liver, pancreas, skeletal muscle, heart and diaphragm without causing significant histopathological changes. Of note, rAAVrh.10 showed excellent muscle transduction following IP administration, highlighting its potential as a new muscle-targeting vector. Nature Publishing Group 2017-01-09 /pmc/articles/PMC5220346/ /pubmed/28067312 http://dx.doi.org/10.1038/srep40336 Text en Copyright © 2017, The Author(s) http://creativecommons.org/licenses/by/4.0/ This work is licensed under a Creative Commons Attribution 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/
spellingShingle Article
Ai, Jianzhong
Li, Jia
Gessler, Dominic J.
Su, Qin
Wei, Qiang
Li, Hong
Gao, Guangping
Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery
title Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery
title_full Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery
title_fullStr Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery
title_full_unstemmed Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery
title_short Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery
title_sort adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5220346/
https://www.ncbi.nlm.nih.gov/pubmed/28067312
http://dx.doi.org/10.1038/srep40336
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