Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library

被引：50

作者：

Ying, Y. ^{[1
]}

Mueller, O. J. ^{[2
]}

Goehringer, C. ^{[2
]}

Leuchs, B. ^{[1
]}

Trepel, M. ^{[3
]}

Katus, H. A. ^{[2
]}

Kleinschmidt, J. A. ^{[1
]}

机构：

[1] Deutsch Krebsforschungszentrum, German Canc Res Ctr, Dept Tumorvirol, D-69120 Heidelberg, Germany

[2] Univ Heidelberg, Heidelberg, Germany

[3] Univ Med Ctr Hamburg Eppendorf, Dept Hematol & Oncol, Hamburg, Germany

来源：

GENE THERAPY | 2010年 / 17卷 / 08期

关键词：

AAV; vector targeting; heart; ADENOASSOCIATED VIRUS TYPE-2; CARDIAC GENE-TRANSFER; AAV2 CAPSID GENE; DIRECTED EVOLUTION; THERAPY VECTOR; TROPISM; TRANSDUCTION; EFFICIENT; EXPRESSION; PROTEIN;

D O I：

10.1038/gt.2010.44

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Selection of targeted vectors from virus display peptide libraries is a versatile and efficient approach to improve vector specificity and efficiency. This strategy has been used to target various cell types in vitro. Here, we report the screening of an adeno-associated virus type 2 (AAV2) display peptide library in vivo to select vectors specifically homing to heart tissue after systemic application in mice. Selected library clones indicated superior specificity of gene transfer compared with wild-type AAV2, AAV9 and a heparin binding-deficient AAV2 mutant. Such targeted vectors were able to reconstitute expression of delta-sarcoglycan in the heart of adult delta-sarcoglycan knockout mice after systemic gene transfer in vivo, attesting to the therapeutic potential of this approach. Gene Therapy (2010) 17, 980-990; doi: 10.1038/gt.2010.44; published online 15 April 2010

引用

页码：980 / 990

页数：11

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