Gene-based antibody strategies for prion diseases

International Journal of Cell Biology

Volumn , Issue , 2013, Pages

  Cardinale, Alessio ^a   Biocca, Silvia ^b  

^a IRCCS SAN RAFFAELE PISANA   (Italy)

^b UNIVERSITY OF ROME TOR VERGATA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; AMYLOID PROTEIN; CELL PENETRATING PEPTIDE; LAMININ RECEPTOR; LENTIVIRUS VECTOR; ORNITHINE DECARBOXYLASE; RECOMBINANT ANTIBODY; SIGNAL PEPTIDE; SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY;

ALZHEIMER DISEASE; BLOOD BRAIN BARRIER; BRAIN CELL; BRAIN DAMAGE; CELL MEMBRANE; CONFORMATIONAL TRANSITION; DISEASE SEVERITY; GENETIC TRANSDUCTION; GRAY MATTER; HIPPOCAMPUS; HUMAN; HYPOTHALAMUS; IN VITRO STUDY; IN VIVO STUDY; INCUBATION TIME; MAMMAL CELL; MICROGLIA; MICROINJECTION; NEUROBLASTOMA CELL; NONHUMAN; NUCLEAR LOCALIZATION SIGNAL; OLIGOMERIZATION; PARKINSON DISEASE; PRION DISEASE; PRIORITY JOURNAL; PROTEIN MOTIF; PROTEIN PROCESSING; PROTEIN PROTEIN INTERACTION; REVIEW; RHABDOMYOSARCOMA; SCRAPIE; SECRETORY PATHWAY; SEROTYPE; SIGNAL TRANSDUCTION; SURFACE PLASMON RESONANCE; THALAMUS; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; WHITE MATTER;

EID: 84884241359     PISSN: 16878876     EISSN: 16878884     Source Type: Journal    
DOI: 10.1155/2013/710406     Document Type: Review

References (57)

1. Collinge J., Prion diseases of humans and animals: their causes and molecular basis. Annual Review of Neuroscience 2001 24 519 550 2-s2.0-0034916581 10.1146/annurev.neuro.24.1.519
2. Chiti F., Dobson C. M., Protein misfolding, functional amyloid, and human disease. Annual Review of Biochemistry 2006 75 333 366 2-s2.0-33746377894 10.1146/annurev.biochem.75.101304.123901
3. Prusiner S. B., Prions. Proceedings of the National Academy of Sciences of the United States of America 1998 95 23 13363 13383 2-s2.0-0032506187 10.1073/pnas.95.23.13363
4. Horiuchi M., Caughey B., Specific binding of normal prion protein to the scrapie form via a localized domain initiates its conversion to the protease-resistant state. The EMBO Journal 1999 18 12 3193 3203 2-s2.0-0033564204 10.1093/emboj/18.12.3193
5. Supattapone S., What makes a prion infectious? Science 2010 327 5969 1091 1092 2-s2.0-77649205447 10.1126/science.1187790
6. Brazier M. W., Mot A. I., White A. R., Collins S. J., Immunotherapeutic approaches in prion disease: progress, challenges and potential directions. Therapeutic Delivery 2013 4 615 628 10.4155/tde.13.30
7. Li L., Napper S., Cashman N. R., Immunotherapy for prion diseases: opportunities and obstacles. Immunotherapy 2010 2 2 269 282 2-s2.0-77953444683 10.2217/imt.10.3
8. Cardinale A., Biocca S., The potential of intracellular antibodies for therapeutic targeting of protein-misfolding diseases. Trends in Molecular Medicine 2008 14 9 373 380 2-s2.0-50549094090 10.1016/j.molmed.2008.07.004
9. Biocca S., Cattaneo A., Intracellular immunization: antibody targeting to subcellular compartments. Trends in Cell Biology 1995 5 6 248 252 2-s2.0-0028997906 10.1016/S0962-8924(00)89019-4
10. Paganetti P., Calanca V., Galli C., Stefani M., Molinari M., β -site specific intrabodies to decrease and prevent generation of Alzheimer's A β peptide. Journal of Cell Biology 2005 168 6 863 868 2-s2.0-15444374558 10.1083/jcb.200410047
11. Zhou C., Emadi S., Sierks M. R., Messer A., A human single-chain Fv intrabody blocks aberrant cellular effects of overexpressed α -synuclein. Molecular Therapy 2004 10 6 1023 1031 2-s2.0-10344257196 10.1016/j.ymthe.2004. 08.019
12. Ryan D. A., Mastrangelo M. A., Narrow W. C., Sullivan M. A., Federoff H. J., Bowers W. J., A Β -directed single-chain antibody delivery via a serotype-1 AAV vector improves learning behavior and pathology in alzheimer's disease mice. Molecular Therapy 2010 18 8 1471 1481 2-s2.0-77955175395 10.1038/mt.2010.111
13. Southwell A. L., Ko J., Patterson P. H., Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease. Journal of Neuroscience 2009 29 43 13589 13602 2-s2.0-70350543879 10.1523/JNEUROSCI.4286-09.2009
14. Cardinale A., Filesi I., Vetrugno V., Pocchiari M., Sy M.-S., Biocca S., Trapping prion protein in the endoplasmic reticulum impairs PrPC maturation and prevents PrPSc accumulation. Journal of Biological Chemistry 2005 280 1 685 694 2-s2.0-12844260131 10.1074/jbc.M407360200
15. Messer A., Lynch S. M., Butler D. C., Developing intrabodies for the therapeutic suppression of neurodegenerative pathology. Expert Opinion on Biological Therapy 2009 9 9 1189 1197 2-s2.0-68949159773 10.1517/ 14712590903176387
16. Biocca S., Neuberger M. S., Cattaneo A., Expression and targeting of intracellular antibodies in mammalian cells. The EMBO Journal 1990 9 1 101 108 2-s2.0-0025122362
17. Cattaneo A., Biocca S., Intracellular Antibodies: Development and Applications 1997 Berlin, Germany Springer
18. Lo A. S.-Y., Zhu Q., Marasco W. A., Intracellular antibodies (intrabodies) and their therapeutic potential. Handbook of Experimental Pharmacology 2008 181 343 373 2-s2.0-38449115445
19. Cardinale A., Filesi I., Mattei S., Biocca S., Intracellular targeting and functional analysis of single-chain Fv fragments in mammalian cells. Methods 2004 34 2 171 178 2-s2.0-4143134285 10.1016/j.ymeth.2004.04.006
20. Biocca S., Intrabody expression in mammalian cells. Cell Engineering 2011 7 179 195 10.1007/978-94-007-1257-7-9
21. Butler D. C., Messer A., Bifunctional anti-huntingtin proteasome-directed intrabodies mediate efficient degradation of mutant huntingtin exon 1 protein fragments. PLoS One 2011 6 12 2-s2.0-84055182535 10.1371/journal.pone.0029199 e29199
22. Blocca S., Ruberti F., Tafani M., Pierandrei-Amaldi P., Cattaneo A., Redox state of single chain Fv fragments targeted to the endoplasmic reticulum, cytosol and mitochondria. Bio/Technology 1995 13 10 1110 1115 2-s2.0-0028883709
23. Dupont E., Prochiantz A., Joliot A., Identification of a signal peptide for unconventional secretion. Journal of Biological Chemistry 2007 282 12 8994 9000 2-s2.0-34247872880 10.1074/jbc.M609246200
24. Yuan B., Sierks M. R., Intracellular targeting and clearance of oligomeric alpha-synuclein alleviates toxicity in mammalian cells. Neuroscience Letters 2009 459 1 16 18 2-s2.0-65749095912 10.1016/j.neulet.2009.04.046
25. Joshi S. N., Butler D. C., Messer A., Fusion to a highly charged proteasomal retargeting sequence increases soluble cytoplasmic expression and efficacy of diverse anti-synuclein intrabodies. MAbs 2012 4 6 686 693
26. Zhou P., Goldstein S., Devadas K., Tewari D., Notkins A. L., Cells transfected with a non-neutralizing antibody gene are resistant to HIV infection: targeting the endoplasmic reticulum and trans-golgi network. Journal of Immunology 1998 160 3 1489 1496 2-s2.0-0031892153
27. Chesnut J. D., Baytan A. R., Russell M., Chang M.-P., Bernard A., Maxwell I. H., Hoeffler J. P., Selective isolation of transiently transfected cells from a mammalian cell population with vectors expressing a membrane anchored single-chain antibody. Journal of Immunological Methods 1996 193 1 17 27 2-s2.0-0029930769 10.1016/0022-1759(96)00032-4
28. Holliger P., Hudson P. J., Engineered antibody fragments and the rise of single domains. Nature Biotechnology 2005 23 9 1126 1136 2-s2.0-27144432842 10.1038/nbt1142
29. Müller D., Kontermann R. E., Bispecific antibodies for cancer immunotherapy: current perspectives. BioDrugs 2010 24 2 89 98 2-s2.0-77749239974 10.2165/11530960-000000000-00000
30. Jendreyko N., Popkov M., Rader C., Barbas C. F. III, Phenotypic knockout of VEGF-R2 and Tie-2 with an intradiabody reduces tumor growth and angiogenesis in vivo. Proceedings of the National Academy of Sciences of the United States of America 2005 102 23 8293 8298 2-s2.0-20444391361 10.1073/pnas.0503168102
31. Hamers-Casterman C., Atarhouch T., Muyldermans S., Robinson G., Hamers C., Songa E. B., Bendahman N., Hamers R., Naturally occurring antibodies devoid of light chains. Nature 1993 363 6428 446 448 2-s2.0-0027310612 10.1038/363446a0
32. Wesolowski J., Alzogaray V., Reyelt J., Unger M., Juarez K., Urrutia M., Cauerhff A., Danquah W., Rissiek B., Scheuplein F., Schwarz N., Adriouch S., Boyer O., Seman M., Licea A., Serreze D. V., Goldbaum F. A., Haag F., Koch-Nolte F., Single domain antibodies: promising experimental and therapeutic tools in infection and immunity. Medical Microbiology and Immunology 2009 198 3 157 174 2-s2.0-68349117269 10.1007/s00430-009-0116-7
33. Li T., Bourgeois J. P., Celli S., Glacial F., Le Sourd A. M., Mecheri S., Weksler B., Romero I., Couraud P. O., Rougeon F., Lafaye P., Cell-penetrating anti-GFAP VHH and corresponding fluorescent fusion protein VHH-GFP spontaneously cross the blood-brain barrier and specifically recognize astrocytes: application to brain imaging. The FASEB Journal 2012 26 3969 3979
34. Jones D. R., Taylor W. A., Bate C., David M., Tayebi M., A camelid anti-PrP antibody abrogates PrP replication in prion-permissive neuroblastoma cell lines. PloS One 2010 5 3 e9804 2-s2.0-78650069813
35. Skrlj N., Drevensek G., Hudoklin S., Romih R., Curin Serbec V., Dolinar M., Recombinant single-chain antibody with the Trojan peptide penetratin positioned in the linker region enables cargo transfer across the blood-brain barrier. Applied Biochemistry and Biotechnology 2013 169 159 169 10.1007/s12010-012-9962-7
36. Hudson P. J., Souriau C., Engineered antibodies. Nature Medicine 2003 9 1 129 134 2-s2.0-0037235949 10.1038/nm0103-129
37. Hoogenboom H. R., Selecting and screening recombinant antibody libraries. Nature Biotechnology 2005 23 9 1105 1116 2-s2.0-27144431943 10.1038/nbt1126
38. Wuertzer C. A., Sullivan M. A., Qiu X., Federoff H. J., CNS delivery of vectored prion-specific single-chain antibodies felays fisease onset. Molecular Therapy 2008 16 3 481 486 2-s2.0-39849085645 10.1038/sj.mt.6300387
39. Moda F., Vimercati C., Campagnani I., Ruggerone M., Giaccone G., Morbin M., Zentilin L., Giacca M., Zucca I., Legname G., Tagliavini F., Brain delivery of AAV9 expressing an anti-PrP monovalent antibody delays prion disease in mice. Prion 2012 6 383 390
40. Filesi I., Cardinale A., Mattei S., Biocca S., Selective re-routing of prion protein to proteasomes and alteration of its vesicular secretion prevent PrPSc formation. Journal of Neurochemistry 2007 101 6 1516 1526 2-s2.0-34249817866 10.1111/j.1471-4159.2006.04439.x
41. Cardinale A., Biocca S., Combating protein misfolding and aggregation by intracellular antibodies. Current Molecular Medicine 2008 8 1 2 11 2-s2.0-42049083108 10.2174/156652408783565595
42. Gauczynski S., Peyrin J.-M., Haïk S., Leucht C., Hundt C., Rieger R., Krasemann S., Deslys J.-P., Dormont D., Lasmézas C. I., Weiss S., The 37-kDa/67-kDa laminin receptor acts as the cell-surface receptor for the cellular prion protein. The EMBO Journal 2001 20 21 5863 5875 2-s2.0-17944363361 10.1093/emboj/20.21.5863
43. Vetrugno V., Cardinale A., Filesi I., Mattei S., Sy M.-S., Pocchiari M., Biocca S., KDEL-tagged anti-prion intrabodies impair PrP lysosomal degradation and inhibit scrapie infectivity. Biochemical and Biophysical Research Communications 2005 338 4 1791 1797 2-s2.0-27844578208 10.1016/j.bbrc.2005.10. 146
44. Donofrio G., Heppner F. L., Polymenidou M., Musahl C., Aguzzi A., Paracrine inhibition of prion propagation by anti-PrP single-chain Fv miniantibodies. Journal of Virology 2005 79 13 8330 8338 2-s2.0-20744448499 10.1128/JVI.79.13.8330-8338.2005
45. Shimizu Y., Kaku-Ushiki Y., Iwamaru Y., Muramoto T., Kitamoto T., Yokoyama T., Mohri S., Tagawa Y., A novel anti-prion protein monoclonal antibody and its single-chain fragment variable derivative with ability to inhibit abnormal prion protein accumulation in cultured cells. Microbiology and Immunology 2010 54 2 112 121 2-s2.0-76649106715 10.1111/j.1348-0421.2009.00190.x
46. Fujita K., Yamaguchi Y., Mori T., Muramatsu N., Miyamoto T., Yano M., Miyata H., Ootsuyama A., Sawada M., Matsuda H., Kaji R., Sakaguchi S., Effects of a brain-engraftable microglial cell line expressing anti-prion scFv antibodies on survival times of mice infected with scrapie prions. Cellular and Molecular Neurobiology 2011 31 7 999 1008 2-s2.0-84859711766
47. Camapana V., Zentilin L., Mirabile I., Kranjc A., Casanova P., Giacca M., Prusiner S. B., Legname G., Zurzolo C., Development of antibody fragments for immunotherapy of prion diseases. Biochemical Journal 2009 418 3 507 515 2-s2.0-62149145178 10.1042/BJ20081541
48. McCown T. J., Adeno-associated virus (AAV) vectors in the CNS. Current Gene Therapy 2011 11 3 181 188 2-s2.0-79958191163
49. Kaplitt M. G., Feigin A., Tang C., Fitzsimons H. L., Mattis P., Lawlor P. A., Bland R. J., Young D., Strybing K., Eidelberg D., During M. J., 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. The Lancet 2007 369 9579 2097 2105 2-s2.0-34250683023 10.1016/S0140-6736(07)60982-9
50. Marks W. J. Jr., Bartus R. T., Siffert J., Davis C. S., Lozano A., Boulis N., Vitek J., Stacy M., Turner D., Verhagen L., Bakay R., Watts R., Guthrie B., Jankovic J., Simpson R., Tagliati M., Alterman R., Stern M., Baltuch G., Starr P. A., Larson P. S., Ostrem J. L., Nutt J., Kieburtz K., Kordower J. H., Olanow C. W., Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. The Lancet Neurology 2010 9 12 1164 1172 2-s2.0-78349247631 10.1016/S1474-4422(10)70254-4
51. Mandel R. J., CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease. Current Opinion in Molecular Therapeutics 2010 12 2 240 247 2-s2.0-77950838482
52. Lasmézas C. I., Deslys J.-P., Robain O., Jaegly A., Beringue V., Peyrin J.-M., Fournier J.-G., Hauw J.-J., Rossier J., Dormont D., Transmission of the BSE agent to mice in the absence of detectable abnormal prion protein. Science 1997 275 5298 402 405 2-s2.0-0344030333 10.1126/science.275.5298.402
53. Foust K. D., Nurre E., Montgomery C. L., Hernandez A., Chan C. M., Kaspar B. K., Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nature Biotechnology 2009 27 1 59 65 2-s2.0-60149106907 10.1038/nbt.1515
54. Duque S., Joussemet B., Riviere C., Marais T., Dubreil L., Douar A.-M., Fyfe J., Moullier P., Colle M.-A., Barkats M., Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Molecular Therapy 2009 17 7 1187 1196 2-s2.0-67649861393 10.1038/mt.2009.71
55. Zincarelli C., Soltys S., Rengo G., Rabinowitz J. E., Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Molecular Therapy 2008 16 6 1073 1080 2-s2.0-44349170706 10.1038/mt.2008.76
56. Zuber C., Knackmuss S., Rey C., Reusch U., Röttgen P., Fröhlich T., Arnold G. J., Pace C., Mitteregger G., Kretzschmar H. A., Little M., Weiss S., Single chain Fv antibodies directed against the 37 kDa/67 kDa laminin receptor as therapeutic tools in prion diseases. Molecular Immunology 2008 45 1 144 151 2-s2.0-34548331958 10.1016/j.molimm.2007.04.030
57. Zuber C., Mitteregger G., Schuhmann N., Rey C., Knackmuss S., Rupprecht W., Reusch U., Pace C., Little M., Kretzschmar H. A., Hallek M., Büning H., Weiss S., 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. Journal of General Virology 2008 89 8 2055 2061 2-s2.0-50549104374 10.1099/vir.0.83670-0