Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses

Molecular Therapy

Volumn 13, Issue 4, 2006, Pages 683-693

  Work, Lorraine M ^a   Büning, Hildegard ^b   Hunt, Ela ^c   Nicklin, Stuart A ^a   Denby, Laura ^a   Britton, Nicola ^a   Leike, Kristen ^b   Odenthal, Margarete ^b   Drebber, Uta ^b   Hallek, Michael ^b   Baker, Andrew H ^a  

^a UNIVERSITY OF GLASGOW   (United Kingdom)

^b UNIVERSITY OF COLOGNE   (Germany)

^c UNIVERSITY OF GLASGOW   (United Kingdom)

Author keywords

Adeno associated virus; Endothelium; Phage display; Rat; Systemic delivery; Targeting

Indexed keywords

HEPARAN SULFATE; PEPTIDE DERIVATIVE; VIRUS DNA;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN; CONTROLLED STUDY; GENE EXPRESSION PROFILING; GENE TARGETING; IN VIVO STUDY; INJECTION; LUNG ENDOTHELIUM; MALE; NONHUMAN; PARVOVIRUS VECTOR; PHAGE DISPLAY; RAT; TRANSGENE; VASCULAR ENDOTHELIUM; VIRAL GENE DELIVERY SYSTEM; VIRION; VIRUS CAPSID; VIRUS VECTOR;

AMINO ACID SEQUENCE; ANIMALS; BLOTTING, WESTERN; CAPSID; CAPSID PROTEINS; CONSENSUS SEQUENCE; DEPENDOVIRUS; DNA, VIRAL; ENDOTHELIUM, VASCULAR; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC VECTORS; HEPARITIN SULFATE; IMMUNOHISTOCHEMISTRY; LAC OPERON; LIVER; MALE; MUTAGENESIS, INSERTIONAL; PEPTIDE LIBRARY; PEPTIDES; PROTEIN STRUCTURE, SECONDARY; RATS; RATS, INBRED WKY; SEQUENCE HOMOLOGY, AMINO ACID; TIME FACTORS; TRANSGENES; VIRION;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS - 2; ANIMALIA; DNA VIRUSES; MIRIDAE; PARVOVIRUS;

EID: 33645502138     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.11.013     Document Type: Article

References (39)

1. W. Arap et. al. Steps towards mapping the human vasculature by phage display Nat. Med. 8 2002 121-127
2. D. Rajotte et. al. Molecular heterogeneity of the vascular endothelium revealed by in vivo phage display J. Clin. Invest. 102 1998 430-437
3. L. Zhang J.A. Hoffman and E. Ruoslahti Molecular profiling of heart endothelial cells Circulation 112 2005 1601-1611
4. J. Pai and E. Ruoslahti Identification of endothelial genes up-regulated in vivo Gene 347 2005 21-33
5. E. Durr et. al. Direct proteomic mapping of the lung microvascular endothelial cell surface in vivo and in cell culture Nat. Biotechnol. 22 2004 985-992
6. P. Oh et. al. Subtractive proteomic mapping of the endothelial surface in lung and solid tumours for tissue-specific therapy Nature 429 2004 629-635
7. O.J. Muller et. al. Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors Nat. Biotechnol. 21 2003 1040-1046
8. W. Arap R. Pasqualiniand E. Ruoslahti Cancer treatment by targeted drug delivery to tumor vasculature in a mouse model Science 279 1998 377-380
9. W. Arap et. al. Targeting the prostate for destruction through a vascular address Proc. Natl. Acad. Sci. USA 99 2002 1527-1531
10. H.M. Ellerby et. al. Anti-cancer activity of targeted pro-apoptotic peptides Nat. Med. 5 1999 1032-1038
11. E. Koivunen et. al. Tumor targeting with a selective gelatinase inhibitor Nat. Biotechnol. 17 1999 768-774
12. M.G. Kolonin P.K. Saha L. Chan R. Pasqualini and W. Arap Reversal of obesity by targeted ablation of adipose tissue Nat. Med. 10 2004 625-632
13. L. Perabo et. al. In vitro selection of viral vectors with modified tropism: The adeno-associated virus display Mol. Ther. 8 2003 151-157
14. L.G. Melo et. al. Endothelium-targeted gene and cell-based therapies for cardiovascular disease Arterioscler. Thromb. Vasc. Biol. 24 2004 1761-1774
15. S.A. Nicklin and A.H. Baker Tropism-modified adenoviral and adeno-associated viral vectors for gene therapy Curr. Gene Ther. 2 2002 273-293
16. P.N. Reynolds et. al. Combined transductional and transcriptional targeting improves the specificity of transgene expression in vivo Nat. Biotechnol. 19 2001 838-842
17. A. Girod et. al. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2 Nat. Med. 5 1999 1052-1056
18. W. Shi and J.S. Bartlett RGD inclusion in VP3 provides adeno-associated virus type 2 (AAV2)-based vectors with a heparan sulfate-independent cell entry mechanism Mol. Ther. 7 2003 515-525
19. S.J. White et. al. Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors Circulation 109 2004 513-519
20. T. Nakamura et. al. Rescue and propagation of fully retargeted oncolytic measles viruses Nat. Biotechnol. 23 2005 209-214
21. B.W. Wu J. Lu T.K. Gallaher W.F. Anderson and P.M. Cannon Identification of regions in the Moloney murine leukemia virus SU protein that tolerate the insertion of an integrin-binding peptide Virology 269 2000 7-17
22. S.A. Nicklin et. al. Efficient and selective AAV-2 mediated gene transfer directed to human vascular endothelial cells Mol. Ther. 4 2001 174-181
23. K.L. Dishart et. al. Third-generation lentivirus vectors efficiently transduce and phenotypically modify vascular cells: Implications for gene therapy J. Mol. Cell. Cardiol. 35 2003 739-748
24. L. Denby S.A. Nicklin and A.H. Baker Adeno-associated virus (AAV)-7 and -8 poorly transduce vascular endothelial cells and are sensitive to proteasomal degradation Gene Ther. 12 2005 1534-1538
25. D.D. Koeberl I.E. Alexander C.L. Halbert D.W. Russell and A.D. Miller Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors Proc. Natl. Acad. Sci. USA 94 1997 1426-1431
26. P. Gregorevic et. al. Systemic delivery of genes to striated muscles using adeno-associated viral vectors Nat. Med. 10 2004 828-834
27. Z. Wang et. al. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart Nat. Biotechnol. 23 2005 321-328
28. K. Pajusola et. al. Cell-type-specific characteristics modulate the transduction efficiency of adeno-associated virus type 2 and restrain infection of endothelial cells J. Virol. 76 2002 11530-11540
29. L.M. Work et. al. Development of efficient viral vectors selective for vascular smooth muscle cells Mol. Ther. 9 2004 198-208
30. A. Abbott Laboratory animals: The Renaissance rat Nature 428 2004 464-466
31. C. Summerford and R.J. Samulski Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions J. Virol. 72 1998 1438-1445
32. H.J. Schluesener and T. Xianglin Selection of recombinant phages binding to pathological endothelial and tumor cells of rat glioblastoma by in-vivo display J. Neurol. Sci. 224 2004 77-82
33. L.M. Higgins et. al. In vivo phage display to identify M cell-targeting ligands Pharm. Res. 21 2004 695-705
34. A. Kern et. al. Identification of a heparin-binding motif on adeno-associated virus type 2 capsids J. Virol. 77 2003 11072-11081
35. L.M. Work C.G. Nicol L. Denby A.H. Baker In vivo biopanning: A methodological approach to identifying novel targeting ligands J.P. Fennell A.H. Baker Hypertension: Methods and Protocols (Methods in Molecular Medicine) 2005 Humana Press New York 395-414
36. T. Hubbard et. al. Ensembl Nucleic Acids Res. 33 2005 D447-D453
37. R. Apweiler UniProt: The Universal Protein knowledge base Nucleic Acids Res. 32 2004 D115-D119
38. E. Hunt Indexed searching on proteins using a suffix sequoia IEEE Bull. Tech. Comm. Data Eng. 27 2004 24-31
39. B.R. Zeeberg et. al. GoMiner: A resource for biological interpretation of genomic and proteomic data Genome Biol. 4 2003 R28