Development of antibody fragments for immunotherapy of prion diseases

Biochemical Journal

Volumn 418, Issue 3, 2009, Pages 507-515

  Camapana, Vincenza ^a   Zentilin, Lorena ^b   Mirabile, Ilaria ^c   Kranjc, Agata ^c   Casanova, Philippe ^a   Giacca, Mauro ^b   Prusiner, Stanley B ^d   Legname, Giuseppe ^c   Zurzolo, Chiara ^a  

^a INSTITUT PASTEUR   (France)

^b INTERNATIONAL CENTRE FOR GENETIC ENGINEERING AND BIOTECHNOLOGY   (Italy)

^c SISSA   (Italy)

^d UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Adeno associated virus; Cellular prion protein (PrPC); Immunotherapy; Lentivirus; Scrapie prion protein (PrPSc); Single chain variable fragment (scFv)

Indexed keywords

ADENO-ASSOCIATED VIRUS; CELLULAR PRION PROTEIN (PRPC); IMMUNOTHERAPY; LENTIVIRUS; SCRAPIE PRION PROTEIN (PRPSC); SINGLE-CHAIN VARIABLE FRAGMENT (SCFV);

CELL CULTURE; CELL MEMBRANES; GENE TRANSFER; MAMMALS; MODEL STRUCTURES; SCANDIUM;

ANTIBODIES;

ADENOVIRUS VECTOR; LENTIVIRUS VECTOR; SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY; ANTIBODY; PRION PROTEIN; VIRUS FUSION PROTEIN;

ANIMAL CELL; ANTIBODY AFFINITY; ARTICLE; BINDING COMPETITION; CELL LINE; CONTROLLED STUDY; DRUG SCREENING; GENETIC TRANSDUCTION; IMMUNOTHERAPY; NERVE CELL; NONHUMAN; PASSIVE IMMUNIZATION; PRION DISEASE; PRIORITY JOURNAL; PROTEIN PROTEIN INTERACTION; VIRAL GENE DELIVERY SYSTEM; ADENO ASSOCIATED VIRUS; ANIMAL; CELL CULTURE; GENE VECTOR; GENETICS; IMMUNOGLOBULIN VARIABLE REGION; IMMUNOLOGY; LENTIVIRINAE; METHODOLOGY; MOUSE; PRION; SCRAPIE;

ADENO-ASSOCIATED VIRUS; LENTIVIRUS; MAMMALIA; MUS; SCRAPIE PRION;

ANIMALS; ANTIBODIES; CELLS, CULTURED; DEPENDOVIRUS; GENETIC VECTORS; IMMUNOGLOBULIN VARIABLE REGION; IMMUNOTHERAPY; LENTIVIRUS; MICE; PRION DISEASES; PRIONS; PRPC PROTEINS; PRPSC PROTEINS; SCRAPIE; VIRAL FUSION PROTEINS;

EID: 62149145178     PISSN: 02646021     EISSN: 14708728     Source Type: Journal    
DOI: 10.1042/BJ20081541     Document Type: Article

References (42)

1. Prusiner, S. B. (1998) Prions. Proc. Natl. Acad. Sci. U.S.A. 95, 13363-13383
2. Will, R. G., Ironside, J. W., Zeidler, M., Cousens, S. N., Estibeiro, K., Alperovitch, A., Poser, S., Pocchiari, M., Hofman, A. and Smith, P. G. (1996) A new variant of Creutzfeldt-Jakob disease in the UK. Lancet 347, 921-925
3. Horiuchi, M. and Caughey, B. (1999) Specific binding of normal prion protein to the scrapie form via a localized domain initiates its conversion to the protease-resistant state. EMBO J. 18, 3193-3203
4. Bueler, H., Aguzzi, A., Sailer, A., Greiner, R. A., Autenried, P., Aguet, M. and Weissmann, C. (1993) Mice devoid of PrP are resistant to scrapie. Cell 73, 1339-1347
5. Richt, J. A., Kasinathan, P., Hamir, A. N., Castilla, J., Sathiyaseelan, T., Vargas, F., Sathiyaseelan, J., Wu, H., Matsushita, H., Koster, J. et al. (2007) Production of cattle lacking prion protein. Nat. Biotechnol. 25, 132-138
6. Brandner, S., Isenmann, S., Raeber, A., Fischer, M., Sailer, A., Kobayashi, Y., Marino, S., Weissmann, C. and Aguzzi, A. (1996) Normal host prion protein necessary for scrapie-induced neurotoxicity. Nature 379, 339-343
7. Weissmann, C. and Aguzzi, A. (2005) Approaches to therapy of prion diseases. Annu. Rev. Med. 56, 321-344
8. Ludewigs, H., Zuber, C., Vana, K., Nikles, D., Zerr, I. and Weiss, S. (2007) Therapeutic approaches for prion disorders. Expert Rev. Anti Infect. Ther. 5, 613-630
9. Trevitt, C. R. and Collinge, J. (2006) A systematic review of prion therapeutics in experimental models. Brain 129, 2241-2265
10. Crozet, C., Lin, Y. L., Mettling, C., Mourton-Gilles, C., Corbeau, P., Lehmann, S. and Perrier, V. (2004) Inhibition of PrPSc formation by lentiviral gene transfer of PrP containing dominant-negative mutations. J. Cell Sci. 117, 5591-5597
11. Pfeifer, A., Eigenbrod, S., Al-Khadra, S., Hofmann, A., Mitteregger, G., Moser, M., Bertsch, U. and Kretzschmar, H. (2006) Lentivector-mediated RNAi efficiently suppresses prion protein and prolongs survival of scrapie-infected mice. J. Clin. Invest. 116, 3204-3210
12. Peretz, D., Williamson, R. A., Kaneko, K., Vergara, J., Leclerc, E., Schmitt-Ulms, G., Mehlhorn, I. R., Legname, G., Wormald, M. R., Rudd, P. M. et al. (2001) Antibodies inhibit prion propagation and clear cell cultures of prion infectivity. Nature 412, 739-743
13. Heppner, F. L. and Aguzzi, A. (2004) Recent developments in prion immunotherapy. Curr. Opin. Immunol. 16, 594-598
14. Enari, M., Flechsig, E. and Weissmann, C. (2001) Scrapie prion protein accumulation by scrapie-infected neuroblastoma cells abrogated by exposure to a prion protein antibody. Proc. Natl. Acad. Sci. U.S.A. 98, 9295-9299
15. Sigurdsson, E. M., Sy, M. S., Li, R., Scholtzova, H., Kascsak, R. J., Kascsak, R., Carp, R., Meeker, H. C., Frangione, B. and Wisniewski, T. (2003) Anti-prion antibodies for prophylaxis following prion exposure in mice. Neurosci. Lett. 336, 185-187
16. Gabizon, R., McKinley, M. P., Groth, D. and Prusiner, S. B. (1988) Immunoaffinity purification and neutralization of scrapie prion infectivity. Proc. Natl. Acad. Sci. U.S.A. 85, 6617-6621
17. Souan, L., Tal, Y., Felling, Y., Cohen, I. R., Taraboulos, A. and Mor, F. (2001) Modulation of proteinase-K resistant prion protein by prion peptide immunization. Eur. J. Immunol. 31, 2338-2346
18. White, A. R., Enever, P., Tayebi, M., Mushens, R., Linehan, J., Brandner, S., Anstee, D., Collinge, J. and Hawke, S. (2003) Monoclonal antibodies inhibit prion replication and delay the development of prion disease. Nature 422, 80-83
19. Polymenidou, M., Heppner, F. L., Pellicioli, E. C., Urich, E., Miele, G., Braun, N., Wopfner, F., Schatzl, H. M., Becher, B. and Aguzzi, A. (2004) Humoral immune response to native eukaryotic prion protein correlates with anti-prion protection. Proc. Natl. Acad. Sci. U.S.A. 101, Suppl. 2, 14670-14676
20. Solforosi, L., Criado, J. R., McGavern, D. B., Wirz, S., Sanchez-Alavez, M., Sugama, S., DeGiorgio, L. A., Volpe, B. T., Wiseman, E., Abalos, G. et al. (2004) Cross-linking cellular prion protein triggers neuronal apoptosis in vivo. Science 303, 1514-1516
21. Chekhonin, V. P., Ryabukhin, I. A., Zhirkov, Y. A., Kashparov, I. A. and Dmitriyeva, T. B. (1995) Transport of hydrophobized fragments of antibodies through the blood-brain barrier. Neuroreport 7, 129-132
22. Girod, J., Fenart, L., Regina, A., Dehouck, M. P., Hong, G., Scherrmann, J. M., Cecchelli, R. and Roux, F. (1999) Transport of cationized anti-tetanus Fab'2 fragments across an in vitro blood-brain barrier model: involvement of the transcytosis pathway. J. Neurochem. 73, 2002-2008
23. Donofrio, G., Heppner, F. L., Polymenidou, M., Musahl, C. and Aguzzi, A. (2005) Paracrine inhibition of prion propagation by anti-PrP single-chain Fv miniantibodies. J. Virol. 79, 8330-8338
24. Cardinale, A., Filesi, I., Vetrugno, V., Pocchiari, M., Sy, M. S. and Biocca, S. (2005) Trapping prion protein in the endoplasmic reticulum impairs PrPC maturation and prevents PrPSc accumulation. J. Biol. Chem. 280, 685-694
25. Vetrugno, V., Cardinale, A., Filesi, I., Mattei, S., Sy, M. S., Pocchiari, M. and Biocca, S. (2005) KDEL-tagged anti-prion intrabodies impair PrP lysosomal degradation and inhibit scrapie infectivity. Biochem. Biophys. Res. Commun. 338, 1791-1797
26. Zuber, C., Knackmuss, S., Rey, C., Reusch, U., Rottgen, P., Frohlich, T., Arnold, G. J., Pace, C., Mitteregger, G., Kretzschmar, H. A. et al. (2008) Single chain Fv antibodies directed against the 37 kDa/67 kDa laminin receptor as therapeutic tools in prion diseases. Mol. Immunol. 45, 144-151
27. Zuber, C., Mitteregger, G., Schuhmann, N., Rey, C., Knackmuss, S., Rupprecht, W., Reusch, U., Pace, C., Little, M., Kretzschmar, H. A. et al. (2008) Delivery of single-chain antibodies (scFvs) directed against the 37/67 kDa laminin receptor into mice via recombinant adeno-associated viral vectors for prion disease gene therapy. J. Gen. Virol. 89, 2055-2061
28. Wuertzer, C. A., Sullivan, M. A., Qiu, X. and Federoff, H. J. (2008) CNS delivery of vectored prion-specific single-chain antibodies delays disease onset. Mol. Ther. 16, 481-486
29. Genoud, N., Ott, D., Braun, N., Prinz, M., Schwarz, P., Suter, U., Trono, D. and Aguzzi, A. (2008) Antiprion prophylaxis by gene transfer of a soluble prion antagonist. Am. J. Pathol. 172, 1287-1296
30. Legname, G., Nelken, P., Guan, Z., Kanyo, Z. F., DeArmond, S. J. and Prusiner, S. B. (2002) Prion and doppel proteins bind to granule cells of the cerebellum. Proc. Natl. Acad. Sci. U.S.A. 99, 16285-16290
31. Zentilin, L., Marcello, A. and Giacca, M. (2001) Involvement of cellular double-stranded DNA break binding proteins in processing of the recombinant adeno-associated virus genome. J. Virol. 75, 12279-12287
32. Williamson, R. A., Peretz, D., Pinilla, C., Ball, H., Bastidas, R. B., Rozenshteyn, R., Houghten, R. A., Prusiner, S. B. and Burton, D. R. (1998) Mapping the prion protein using recombinant antibodies. J. Virol. 72, 9413-9418
33. Berman, H. M., Westbrook, J., Feng, Z., Gilliland, G., Bhat, T. N., Weissig, H., Shindyalov, I. N. and Bourne, P. E. (2000) The protein data bank. Nucleic Acids Res. 28, 235-242
34. Luginbuhl, B., Kanyo, Z., Jones, R. M., Fletterick, R. J., Prusiner, S. B., Cohen, F. E., Williamson, R. A., Burton, D. R. and Pluckthun, A. (2006) Directed evolution of an anti-prion protein scFv fragment to an affinity of 1 pM and its structural interpretation. J. Mol. Biol. 363, 75-97
35. Sali, A. and Blundell, T. L. (1993) Comparative protein modelling by satisfaction of spatial restraints. J. Mol. Biol. 234, 779-815
36. Calzolai, L. and Zahn, R. (2003) Influence of pH on NMR structure and stability of the human prion protein globular domain. J. Biol. Chem. 278, 35592-35596
37. de Vries, S. J., van Dijk, A. D., Krzeminski, M., van Dijk, M., Thureau, A., Hsu, V., Wassenaar, T. and Bonvin, A. M. (2007) HADDOCK versus HADDOCK: new features and performance of HADDOCK2.0 on the CAPRI targets. Proteins 69, 726-733
38. Dominguez, C., Boelens, R. and Bonvin, A. M. (2003) HADDOCK: a protein-protein docking approach based on biochemical or biophysical information. J. Am. Chem. Soc. 125, 1731-1737
39. Ross, T. M., Martinez, P. M., Renner, J. C., Thorne, R. G., Hanson, L. R. and Frey, 2nd, W. H. (2004) Intranasal administration of interferon beta bypasses the blood-brain barrier to target the central nervous system and cervical lymph nodes: a non-invasive treatment strategy for multiple sclerosis. J. Neuroimmunol. 151, 66-77
40. Thorne, R. G., Hanson, L. R., Ross, T. M., Tung, D. and Frey, 2nd, W. H. (2008) Delivery of interferon-beta to the monkey nervous system following intranasal administration. Neuroscience 152, 785-797
41. Kaplitt, M. G., Feigin, A., Tang, C., Fitzsimons, H. L., Mattis, P., Lawlor, P. A., Bland, R. J., Young, D., Strybing, K., Eidelberg, D. and During, M. J. (2007) Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369, 2097-2105
42. Horonchik, L., Tzaban, S., Ben-Zaken, O., Yedidia, Y., Rouvinski, A., Papy-Garcia, D., Barritault, D., Vlodavsky, I. and Taraboulos, A. (2005) Heparan sulfate is a cellular receptor for purified infectious prions. J. Biol. Chem. 280, 17062-17067