Generating differentially targeted amyloid-β specific intrabodies as a passive vaccination strategy for alzheimer's disease

Molecular Therapy

Volumn 17, Issue 12, 2009, Pages 2031-2040

  Sudol, Kelly L ^a   Mastrangelo, Michael A ^a   Narrow, Wade C ^a   Frazer, Maria E ^a   Levites, Yona R ^b   Golde, Todd E ^b   Federoff, Howard J ^a,c   Bowers, William J ^a  

^a UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^b MAYO CLINIC   (United States)

^c GEORGETOWN UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMYLOID BETA PROTEIN; AMYLOID BETA PROTEIN ANTIBODY; AMYLOID BETA PROTEIN[1-42]; AMYLOID PRECURSOR PROTEIN; DOXYCYCLINE; EPITOPE; MUTANT PROTEIN; PARVOVIRUS VECTOR; SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY; TAU PROTEIN; THREONINE;

ALZHEIMER DISEASE; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY SPECIFICITY; ARTICLE; BIOENGINEERING; CONTROLLED STUDY; DISEASE COURSE; DRUG EFFICACY; DRUG TARGETING; ENDOPLASMIC RETICULUM; FEMALE; GENETIC TRANSCRIPTION; MALE; MOUSE; NONHUMAN; VACCINATION;

ADENO-ASSOCIATED VIRUS; MUS;

EID: 73849121797     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.174     Document Type: Article

References (46)

1. Masters, CL, Multhaup, G, Simms, G, Pottgiesser, J, Martins, RN and Beyreuther, K (1985). Neuronal origin of a cerebral amyloid: neurofbrillary tangles of Alzheimer's disease contain the same protein as the amyloid of plaque cores and blood vessels. EMBO J 4: 2757-2763.
2. Chui, DH, Dobo, E, Makifuchi, T, Akiyama, H, Kawakatsu, S, Petit, A et al. (2001). Apoptotic neurons in Alzheimer's disease frequently show intracellular Abeta42 labeling. J Alzheimers Dis 3: 231-239.
3. Kienlen-Campard, P, Miolet, S, Tasiaux, B and Octave, JN (2002). Intracellular amyloid-beta 1-42, but not extracellular soluble amyloid-beta peptides, induces neuronal apoptosis. J Biol Chem 277: 15666-15670.
4. LaFerla, FM, Tinkle, BT, Bieberich, CJ, Haudenschild, CC and Jay, G (1995). The Alzheimer's A beta peptide induces neurodegeneration and apoptotic cell death in transgenic mice. Nat Genet 9: 21-30.
5. Takeda, K, Araki, W and Tabira, T (2004). Enhanced generation of intracellular Abeta42 amyloid peptide by mutation of presenilins PS1 and PS2. Eur J Neurosci 19: 258-264.
6. Misonou, H, Morishima-Kawashima, M and Ihara, Y (2000). Oxidative stress induces intracellular accumulation of amyloid beta-protein (Abeta) in human neuroblastoma cells. Biochemistry 39: 6951-6959.
7. Levites, Y, Jansen, K, Smithson, LA, Dakin, R, Holloway, VM, Das, P et al. (2006). Intracranial adeno-associated virus-mediated delivery of anti-pan amyloid beta, amyloid beta40, and amyloid beta42 single-chain variable fragments attenuates plaque pathology in amyloid precursor protein mice. J Neurosci 26: 11923-11928.
8. Holliger, P and Hudson, PJ (2005). Engineered antibody fragments and the rise of single domains. Nat Biotechnol 23: 1126-1136.
9. Zhu, Q, Zeng, C, Huhalov, A, Yao, J, Turi, TG, Danley, D et al. (1999). Extended half-life and elevated steady-state level of a single-chain Fv intrabody are critical for specifc intracellular retargeting of its antigen, caspase-7. J Immunol Methods 231: 207-222.
10. Lobato, MN and Rabbitts, TH (2003). Intracellular antibodies and challenges facing their use as therapeutic agents. Trends Mol Med 9: 390-396.
11. Richardson, JH and Marasco, WA (1995). Intracellular antibodies: development and therapeutic potential. Trends Biotechnol 13: 306-310.
12. Miller, TW and Messer, A (2005). Intrabody applications in neurological disorders: progress and future prospects. Mol Ther 12: 394-401.
13. Fukuchi, K, Kamino, K, Deeb, SS, Furlong, CE, Sundstrom, JA, Smith, AC et al. (1992). Expression of a carboxy-terminal region of the beta-amyloid precursor protein in a heterogeneous culture of neuroblastoma cells: evidence for altered processing and selective neurotoxicity. Brain Res Mol Brain Res 16: 37-46.
14. Strathdee, CA, McLeod, MR and Hall, JR (1999). Effcient control of tetracycline-responsive gene expression from an autoregulated bi-directional expression vector. Gene 229: 21-29.
15. Cook, DG, Forman, MS, Sung, JC, Leight, S, Kolson, DL, Iwatsubo, T et al. (1997). Alzheimer's A beta(1-42) is generated in the endoplasmic reticulum/intermediate compartment of NT2N cells. Nat Med 3: 1021-1023.
16. Hare, JF (2006). Intracellular pathways of folded and misfolded amyloid precursor protein degradation. Arch Biochem Biophys 451: 79-90.
17. Selkoe, DJ (1997). Alzheimer's disease: genotypes, phenotypes, and treatments. Science 275: 630-631.
18. Selkoe, DJ (1998). The cell biology of beta-amyloid precursor protein and presenilin in Alzheimer's disease. Trends Cell Biol 8: 447-453.
19. Wisniewski, T, Ghiso, J and Frangione, B (1997). Biology of A beta amyloid in Alzheimer's disease. Neurobiol Dis 4: 313-328. (Pubitemid 28018660)
20. Younkin, SG (1998). The role of A beta 42 in Alzheimer's disease. J Physiol Paris 92: 289-292.
21. Oddo, S, Caccamo, A, Kitazawa, M, Tseng, BP and LaFerla, FM (2003). Amyloid deposition precedes tangle formation in a triple transgenic model of Alzheimer's disease. Neurobiol Aging 24: 1063-1070.
22. Oddo, S, Caccamo, A, Shepherd, JD, Murphy, MP, Golde, TE, Kayed, R et al. (2003). Triple-transgenic model of Alzheimer's disease with plaques and tangles: intracellular Abeta and synaptic dysfunction. Neuron 39: 409-421.
23. Mastrangelo, MA and Bowers, WJ (2008). Detailed immunohistochemical characterization of temporal and spatial progression of Alzheimer's disease-related pathologies in male triple-transgenic mice. BMC Neurosci 9: 81.
24. Billings, LM, Oddo, S, Green, KN, McGaugh, JL and LaFerla, FM (2005). Intraneuronal Abeta causes the onset of early Alzheimer's disease-related cognitive defcits in transgenic mice. Neuron 45: 675-688.
25. Hoe, HS and Rebeck, GW (2008). Functional interactions of APP with the apoE receptor family. J Neurochem 106: 2263-2271.
26. Frazer, ME, Hughes, JE, Mastrangelo, MA, Tibbens, JL, Federoff, HJ and Bowers, WJ (2008). Reduced pathology and improved behavioral performance in Alzheimer's disease mice vaccinated with HSV amplicons expressing amyloid-beta and interleukin-4. Mol Ther 16: 845-853.
27. Oddo, S, Billings, L, Kesslak, J P, Cribbs, DH and LaFerla, FM (2004). Abeta immunotherapy leads to clearance of early, but not late, hyperphosphorylated tau aggregates via the proteasome. Neuron 43: 321-332.
28. Oddo, S, Caccamo, A, Tran, L, Lambert, M P, Glabe, CG, Klein, WL et al. (2006). Temporal profle of amyloid-beta (Abeta) oligomerization in an in vivo model of Alzheimer disease. A link between Abeta and tau pathology. J Biol Chem 281: 1599-1604.
29. Fukuchi, K, Accavitti-Loper, MA, Kim, HD, Tahara, K, Cao, Y, Lewis, TL et al. (2006). Amelioration of amyloid load by anti-Abeta single-chain antibody in Alzheimer mouse model. Biochem Biophys Res Commun 344: 79-86.
30. Fukuchi, K, Tahara, K, Kim, HD, Maxwell, JA, Lewis, TL, Accavitti-Loper, MA et al. (2006). Anti-Abeta single-chain antibody delivery via adeno-associated virus for treatment of Alzheimer's disease. Neurobiol Dis 23: 502-511.
31. Lecerf, JM, Shirley, TL, Zhu, Q, Kazantsev, A, Amersdorfer, P, Housman, DE et al. (2001). Human single-chain Fv intrabodies counteract in situ huntingtin aggregation in cellular models of Huntington's disease. Proc Natl Acad Sci USA 98: 4764-4769.
32. Paganetti, P, Calanca, V, Galli, C, Stefani, M and Molinari, M (2005). beta-site specific intrabodies to decrease and prevent generation of Alzheimer's Abeta peptide. J Cell Biol 168: 863-868.
33. Lynch, SM, Zhou, C and Messer, A (2008). An scFv intrabody against the nonamyloid component of alpha-synuclein reduces intracellular aggregation and toxicity. J Mol Biol 377: 136-147.
34. Skovronsky, DM, Doms, RW and Lee, VM (1998). Detection of a novel intraneuronal pool of insoluble amyloid beta protein that accumulates with time in culture. J Cell Biol 141: 1031-1039.
35. Cardinale, A, Filesi, I, Mattei, S and Biocca, S (2003). Evidence for proteasome dysfunction in cytotoxicity mediated by anti-Ras intracellular antibodies. Eur J Biochem 270: 3389-3397.
36. Shumway, SD, Maki, M and Miyamoto, S (1999). The PEST domain of IkappaBalpha is necessary and suffcient for in vitro degradation by mu-calpain. J Biol Chem 274: 30874-30881. (Pubitemid 129576835)
37. Johnston, J, Tazelaar, J, Rivera, VM, Clackson, T, Gao, GP and Wilson, JM (2003). Regulated expression of erythropoietin from an AAV vector safely improves the anemia of beta-thalassemia in a mouse model. Mol Ther 7: 493-497.
38. Haberman, RP and McCown, TJ (2002). Regulation of gene expression in adeno-associated virus vectors in the brain. Methods 28: 219-226.
39. Bobo, RH, Laske, DW, Akbasak, A, Morrison, PF, Dedrick, RL and Oldfeld, EH (1994). Convection-enhanced delivery of macromolecules in the brain. Proc Natl Acad Sci USA 91: 2076-2080.
40. Lidar, Z, Mardor, Y, Jonas, T, Pfeffer, R, Faibel, M, Nass, D et al. (2004). Convection-enhanced delivery of paclitaxel for the treatment of recurrent malignant glioma: a phase I/II clinical study. J Neurosurg 100: 472-479.
41. Kunwar, S (2003). Convection enhanced delivery of IL13-PE38QQR for treatment of recurrent malignant glioma: presentation of interim fndings from ongoing phase 1 studies. Acta Neurochir Suppl 88: 105-111.
42. Fiandaca, MS, Varenika, V, Eberling, J, McKnight, T, Bringas, J, Pivirotto, P et al. (2008). Real-time MR imaging of adeno-associated viral vector delivery to the primate brain. Neuroimage. (epub a head of print).
43. Janelsins, MC, Mastrangelo, MA, Park, KM, Sudol, KL, Narrow, WC, Oddo, S et al. (2008). Chronic neuron-specific tumor necrosis factor-alpha expression enhances the local infammatory environment ultimately leading to neuronal death in 3xTg-AD mice. Am J Pathol 173: 1768-1782.
44. Urabe, M, Ding, C and Kotin, RM (2002). Insect cells as a factory to produce adeno-associated virus type 2 vectors. Hum Gene Ther 13: 1935-1943.
45. Wuertzer, CA, Sullivan, MA, Qiu, X and Federoff, HJ (2008). CNS delivery of vectored prion-specific single-chain antibodies delays disease onset. Mol Ther 16: 481-486.
46. Janelsins, MC, Mastrangelo, MA, Oddo, S, LaFerla, FM, Federoff, HJ and Bowers, WJ (2005). Early correlation of microglial activation with enhanced tumor necrosis factor-alpha and monocyte chemoattractant protein-1 expression specifically within the entorhinal cortex of triple transgenic Alzheimer's disease mice. J Neuroinfammation 2: 23.