UMMS Affiliation

Horae Gene Therapy Center; Department of Microbiology and Physiological Systems

Publication Date

1-9-2017

Document Type

Article

Disciplines

Genetics and Genomics | Therapeutics

Abstract

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.

Rights and Permissions

Copyright © 2017, The Author(s). Citation: Sci Rep. 2017 Jan 9;7:40336. doi: 10.1038/srep40336. Link to article on publisher's site

DOI of Published Version

10.1038/srep40336

Related Resources

Link to Article in PubMed

Keywords

Applied microbiology, Gene therapy, Genetic transduction, Translational research

Journal/Book/Conference Title

Scientific reports

PubMed ID

28067312

Creative Commons License

Creative Commons Attribution 4.0 License
This work is licensed under a Creative Commons Attribution 4.0 License.

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