Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6

Journal of Virology

Volumn 86, Issue 12, 2012, Pages 6620-6631

  Denard, Jerome ^a   Beley, Cyriaque ^b   Kotin, Robert ^c   Lai Kuen, René ^d   Blot, Stéphane ^b,e   Leh, Hervé ^f   Asokan, Aravind ^g   Jude Samulski, R ^g   Moullier, Philippe ^a,h,i   Voit, Thomas ^b   Garcia, Luis ^b   Svinartchouk, Fedor ^a  

^a GÉNÉTHON   (France)

^b SORBONNE UNIVERSITÉ   (France)

^c NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^d UNIVERSITÉ PARIS DESCARTES   (France)

^e UNIVERSITÉ PARIS EST CRÉTEIL   (France)

^f Ecole Normale Superieure de Cachan   (France)

^g UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^h UNIVERSITÉ DE NANTES   (France)

ⁱ UNIVERSITY OF FLORIDA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BINDING PROTEIN; C REACTIVE PROTEIN; GALECTIN 3 BINDING PROTEIN; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ADULT; ANTIBODY AFFINITY; ARTICLE; BINDING AFFINITY; BLOOD ANALYSIS; COMPLEX FORMATION; HUMAN; HUMAN ADENOVIRUS 2; INNATE IMMUNITY; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN AGGREGATION; PROTEIN BLOOD LEVEL; PROTEIN INTERACTION; PROTEIN LOCALIZATION; SCREENING TEST; SEROTYPE; SPECIES DIFFERENCE; VIRUS PURIFICATION;

ANIMALS; ANTIGENS, NEOPLASM; CARRIER PROTEINS; DEPENDOVIRUS; DOGS; GENE THERAPY; GENETIC VECTORS; GLYCOPROTEINS; HUMANS; MACACA; MICE; MICE, INBRED C57BL; PROTEIN BINDING; SPECIES SPECIFICITY; TRANSDUCTION, GENETIC; TUMOR MARKERS, BIOLOGICAL;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 6; ANIMALIA; CANIS FAMILIARIS; MACACA; MUS;

EID: 84864008603     PISSN: 0022538X     EISSN: 10985514     Source Type: Journal    
DOI: 10.1128/JVI.00297-12     Document Type: Article

References (52)

1. Abernethy TJ, Avery OT. 1941. The occurrence during acute infections of a protein not normally present in the blood. I. Distribution of the reactive protein in patients' sera and the effect of calcium on the flocculation reaction with C polysaccharide of pneumococcus. J. Exp. Med. 73:173-182.
2. Agrawal A, Singh PP, Bottazzi B, Garlanda C, Mantovani A. 2009. Pattern recognition by pentraxins. Adv. Exp. Med. Biol. 653:98 -116.
3. Asokan A, et al. 2010. Reengineering a receptor footprint of adenoassociated virus enables selective and systemic gene transfer to muscle. Nat. Biotechnol. 28:79-82.
4. Bish LT, et al. 2012. Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping. Mol. Ther. 20:580 -589.
5. Boutin S, et al. 2010. Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors. Hum. Gene Ther. 21:704 -712.
6. Boye SE, et al. 2010. Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. PLoS One 5:e11306. doi:10.1371/journal.pone.0011306.
7. Brantly ML, et al. 2009. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc. Natl. Acad. Sci. U. S. A. 106:16363-16368.
8. Chenail G, Brown NE, Shea A, Feire AL, Deng G. 2011. Real-time analysis of antibody interactions with whole enveloped human cytomegalovirus using surface plasmon resonance. Anal. Biochem. 411:58-63.
9. Chicheportiche Y, Vassalli P. 1994. Cloning and expression of a mouse macrophage cDNA coding for a membrane glycoprotein of the scavenger receptor cysteine-rich domain family. J. Biol. Chem. 269:5512-5517.
10. Denard J, et al. 2009. Quantitative proteomic analysis of lentiviral vectors using 2-DE. Proteomics 9:3666 -3676.
11. Denti MA, et al. 2006. Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model. Proc. Natl. Acad. Sci. U. S. A. 103: 3758-3763.
12. DiPrimio N, McPhee SW, Samulski RJ. 2010. Adeno-associated virus for the treatment of muscle diseases: toward clinical trials. Curr. Opin. Mol. Ther. 12:553-560.
13. Drittanti L, Rivet C, Manceau P, Danos O, Vega M. 2000. High throughput production, screening and analysis of adeno-associated viral vectors. Gene Ther. 7:924 -929.
14. Friedman J, Trahey M, Weissman I. 1993. Cloning and characterization of cyclophilin C-associated protein: a candidate natural cellular ligand for cyclophilin C. Proc. Natl. Acad. Sci. U. S. A. 90:6815- 6819.
15. Garlanda C, Bottazzi B, Bastone A, Mantovani A. 2005. Pentraxins at the crossroads between innate immunity, inflammation, matrix deposition, and female fertility. Annu. Rev. Immunol. 23:337-366.
16. Goyenvalle A, et al. 2004. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 306:1796 -1799.
17. Gregorevic P, Blankinship MJ, Allen JM, Chamberlain JS. 2008. Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice. Mol. Ther. 16:657- 664.
18. Gregorevic P, et al. 2004. Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat. Med. 10:828-834.
19. Groschel B, et al. 2000. Elevated plasma levels of 90K (Mac-2 BP) immunostimulatory glycoprotein in HIV-1-infected children. J. Clin. Immunol. 20:117-122.
20. Hasbrouck NC, High KA. 2008. AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects. Gene Ther. 15:870-875.
21. Hellstern S, et al. 2002. Functional studies on recombinant domains of Mac-2-binding protein. J. Biol. Chem. 277:15690 -15696.
22. Hermens WT, et al. 1999. Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system. Hum. Gene Ther. 10:1885-1891.
23. Hohenester E, Sasaki T, Timpl R. 1999. Crystal structure of a scavenger receptor cysteine-rich domain sheds light on an ancient superfamily. Nat. Struct. Biol. 6:228 -232.
24. Jalkanen K, et al. 2001. Distinct ligand binding properties of Mac-2-binding protein and mouse cyclophilin [correction of mousephilin] C-associated protein. Eur. J. Immunol. 31:3075-3084.
25. Kornegay JN, et al. 2010. Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs. Mol. Ther. 18:1501-1508.
26. Koths K, Taylor E, Halenbeck R, Casipit C, Wang A. 1993. Cloning and characterization of a human Mac-2-binding protein, a new member of the superfamily defined by the macrophage scavenger receptor cysteine-rich domain. J. Biol. Chem. 268:14245-14249.
27. Li H, et al. 2009. A preclinical animal model to assess the effect of preexisting immunity on AAV-mediated gene transfer. Mol. Ther. 17:1215- 1224.
28. Lillicrap D, VandenDriessche T, High K. 2006. Cellular and genetic therapies for haemophilia. Haemophilia 12(Suppl. 3):36-41.
29. Lorain S, et al. 2008. Transient immunomodulation allows repeated injections of AAV1 and correction of muscular dystrophy in multiple muscles. Mol. Ther. 16:541-547.
30. Mingozzi F, High KA. 2007. Immune responses to AAV in clinical trials. Curr. Gene Ther. 7:316 -324.
31. Muller SA, et al. 1999. Domain organization of Mac-2 binding protein and its oligomerization to linear and ring-like structures. J. Mol. Biol. 291:801- 813.
32. Ng R, et al. 2010. Structural characterization of the dual glycan binding adeno-associated virus serotype 6. J. Virol. 84:12945-12957.
33. Nogues C, et al. 2010. Characterisation of peptide microarrays for studying antibody-antigen binding using surface plasmon resonance imagery. PLoS One 5:e12152. doi:10.1371/journal.pone.0012152.
34. Odom GL, Gregorevic P, Allen JM, Chamberlain JS. 2010. Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6. Mol. Ther. 19:36-45.
35. Odom GL, Gregorevic P, Allen JM, Finn E, Chamberlain JS. 2008. Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice. Mol. Ther. 16:1539 -1545.
36. Pang J, et al. 2010. Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency. Gene Ther. 17:815- 826.
37. Peiser L, Mukhopadhyay S, Gordon S. 2002. Scavenger receptors in innate immunity. Curr. Opin. Immunol. 14:123-128.
38. Penaud-Budloo M, et al. 2008. Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle. J. Virol. 82:7875-7885.
39. Polyak S, et al. 2008. Gene delivery to intestinal epithelial cells in vitro and in vivo with recombinant adeno-associated virus types 1, 2 and 5. Dig. Dis. Sci. 53:1261-1270.
40. Rapti K, et al. 2012. Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models. Mol. Ther. 20:73- 83.
41. Resnick D, Pearson A, Krieger M. 1994. The SRCR superfamily: a family reminiscent of the Ig superfamily. Trends Biochem. Sci. 19:5- 8.
42. Sasaki T, Brakebusch C, Engel J, Timpl R. 1998. Mac-2 binding protein is a cell-adhesive protein of the extracellular matrix which self-assembles into ring-like structures and binds beta1 integrins, collagens and fibronectin. EMBO J. 17:1606 -1613.
43. Siboo R, Kulisek E. 1978. A fluorescent immunoassay for the quantification of C-reactive protein. J. Immunol. Methods 23:59-67.
44. Smith RH, Levy JR, Kotin RM. 2009. A simplified baculovirus-AAV expression vector system coupled with one-step affinity purification yields high-titer rAAV stocks from insect cells. Mol. Ther. 17:1888 -1896.
45. Stone D, et al. 2008. Biodistribution and safety profile of recombinant adeno-associated virus serotype 6 vectors following intravenous delivery. J. Virol. 82:7711-7715.
46. Towne C, Raoul C, Schneider BL, Aebischer P. 2008. Systemic AAV6 delivery mediatingRNAinterference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice. Mol. Ther. 16: 1018-1025.
47. van der Laan LJ, Wang Y, Tilanus HW, Janssen HL, Pan Q. 2011. AAV-mediated gene therapy for liver diseases: the prime candidate for clinical application? Expert Opin. Biol. Ther. 11:315-327.
48. Waddington SN, et al. 2008. Adenovirus serotype 5 hexon mediates liver gene transfer. Cell 132:397- 409.
49. Wang M, et al. 2001. MUSEAP, a novel reporter gene for the study of long-term gene expression in immunocompetent mice. Gene 279:99 -108.
50. Wright JF. 2009. Transient transfection methods for clinical adenoassociated viral vector production. Hum. Gene Ther. 20:698 -706.
51. Wu Z, et al. 2006. Single amino acid changes can influence titer, heparin binding, and tissue tropism in different adeno-associated virus serotypes. J. Virol. 80:11393-11397.
52. Yue Y, et al. 2008. A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol. Ther. 16:1944 -1952.