Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library
Standard
Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library. / Ying, Y; Müller, O J; Goehringer, C; Leuchs, B; Trepel, M; Katus, H A; Kleinschmidt, J A.
in: GENE THER, Jahrgang 17, Nr. 8, 08.2010, S. 980-90.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
Harvard
APA
Vancouver
Bibtex
}
RIS
TY - JOUR
T1 - Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library
AU - Ying, Y
AU - Müller, O J
AU - Goehringer, C
AU - Leuchs, B
AU - Trepel, M
AU - Katus, H A
AU - Kleinschmidt, J A
PY - 2010/8
Y1 - 2010/8
N2 - 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.
AB - 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.
KW - Animals
KW - Cell Line
KW - Dependovirus
KW - Gene Transfer Techniques
KW - Genetic Therapy
KW - Genetic Vectors
KW - Mice
KW - Myocardium
KW - Myocytes, Cardiac
KW - Peptide Library
KW - Rats
KW - Sarcoglycans
KW - Transduction, Genetic
KW - Journal Article
KW - Research Support, Non-U.S. Gov't
U2 - 10.1038/gt.2010.44
DO - 10.1038/gt.2010.44
M3 - SCORING: Journal article
C2 - 20393510
VL - 17
SP - 980
EP - 990
JO - GENE THER
JF - GENE THER
SN - 0969-7128
IS - 8
ER -