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Sci Rep. 2017 Jan 9;7:40336. doi: 10.1038/srep40336.

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

Ai J1,2,3, Li J2, Gessler DJ2,3, Su Q2, Wei Q1, Li H1, Gao G2,3,4.

Author information

1
Institute of Urology, Department of Urology, West China Hospital, Sichuan University, Chengdu, Sichuan, P.R. China.
2
Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
3
Department of Microbiology and Physiology Systems, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
4
State Key Laboratory of Biotherapy, West China Hospital, Sichuan University, Chengdu, P.R. China.

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.

PMID:
28067312
PMCID:
PMC5220346
DOI:
10.1038/srep40336
[Indexed for MEDLINE]
Free PMC Article

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