Immunization in familial amyloidotic polyneuropathy: Counteracting deposition by immunization with a Y78F TTR mutant

Laboratory Investigation

Volumn 86, Issue 1, 2006, Pages 23-31

  Terazaki, Hisayasu ^a,b   Ando, Yukio ^b   Fernandes, Rui ^a   Yamamura, Ken Ichi ^b   Maeda, Shuichiro ^c   Saraiva, Maria J ^a,d  

^a UNIVERSITY OF PORTO   (Portugal)

^b KUMAMOTO UNIVERSITY   (Japan)

^c UNIVERSITY OF YAMANASHI   (Japan)

^d UNIVERSITY OF PORTO   (Portugal)

Author keywords

Alzheimer disease; Amyloid; Familial amyloidotic polyneuropathy; Immunization; Transthyretin

Indexed keywords

MUTANT PROTEIN; PREALBUMIN; PROTEIN V30M; PROTEIN Y78F; UNCLASSIFIED DRUG;

AMINO ACID SEQUENCE; AMINO ACID SUBSTITUTION; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY RESPONSE; ARTICLE; BIOACCUMULATION; CELL INFILTRATION; CELLULAR IMMUNITY; CHOROID PLEXUS; CONTROLLED STUDY; FAMILIAL AMYLOID POLYNEUROPATHY; GENE MUTATION; IMMUNIZATION; IMMUNOHISTOCHEMISTRY; LANGERHANS CELL; LIVER; LYMPHOCYTIC INFILTRATION; MACROPHAGE; MOUSE; NONHUMAN; PRIORITY JOURNAL; TRANSGENIC MOUSE;

AMYLOID NEUROPATHIES, FAMILIAL; ANIMALS; ENZYME-LINKED IMMUNOSORBENT ASSAY; MICE; MICE, TRANSGENIC; MUTATION; PREALBUMIN;

EID: 32844473029     PISSN: 00236837     EISSN: 15300307     Source Type: Journal    
DOI: 10.1038/labinvest.3700365     Document Type: Article

References (29)

1. Dobson CM. Protein folding and its links with human disease. Biochem Soc Sym 2001;68:1-26.
2. Selkoe DJ. Amyloid beta-protein and the genetics of Alzheimer's disease. J Biol Chem 1996;271:18295-18298.
3. Weissmann C, Enari M, Klohn PC, et al. Transmission of prions. J Infect Dis 2002;186:157-165.
4. Saraiva MJ. Transthyretin amyloidosis: a tale of weak interactions. FEBS Lett 2002;498:201-203.
5. Andrade C. A peculiar form of peripheral neuropathy; familiar atypical generalized amyloidosis with special involvement of the peripheral nerves. Brain 1952;75:408-427.
6. Ando Y, Araki S, Ando M. Transthyretin and familial amyloidotic polyneuropathy. Intern Med 1993;32:920-922.
7. de Carvalho M, Conceição I, Bentes C, et al. Long-term quantitative evaluation of liver transplantation in familial amyloid polyneuropathy (Portuguese V30M). Amyloid 2002;9:126-133.
8. Suhr OB, Ericzon BG, Friman S. Long-term follow-up of survival of liver transplant recipients with familial amyloid polyneuropathy (Portuguese type). Liver Transpl 2002;8:787-794.
9. Haraoka K, Ando Y, Ando E, et al. Presence of variant transthyretin in aqueous humor of a patient with familial amyloidotic polyneuropathy after liver transplantation. Amyloid 2002;9:247-251.
10. Ando E, Ando Y, Haraoka K. Ocular amyloid involvement after liver transplantation for polyneuropathy. Ann Intern Med 2001;135:931-932.
11. Altland K, Winter P, Saraiva MJ, et al. Sulfite and base for the treatment of familial amyloidotic polyneuropathy: two additive approaches to stabilize the conformation of human amyloidogenic transthyretin. Neurogenetics 2004;8:61-67.
12. Oza VB, Smith C, Raman P, et al. Synthesis, structure, and activity of diclofenac analogues as transthyretin amyloid fibril formation inhibitors. J Med Chem 2002;45:321-332.
13. Cardoso I, Merlini G, Saraiva MJ. 4′-Iodo-4′-deoxydoxorubicin and tetracyclines disrupt transthyretin amyloid fibrils in vitro producing noncytotoxic species: screening for TTR fibril disrupters. FASEB J 2003;17:803-809.
14. Schenk D, Barbour R, Dunn W, et al. Immunization with amyloid-β attenuates Alzheimer-disease-like pathology in the PDAPP mouse. Nature 1999;400:173-177.
15. Bard F, Cannon C, Barbour R, et al. Peripherally administered antibodies against amyloid β-peptide enter the central nervous system and reduce pathology in a mouse model of Alzheimer disease. Nat Med 2000;6:916-919.
16. Sigurdsson EM, Brown DR, Daniels M, et al. Immunization delays the onset of prion disease in mice. Am J Pathol 2002;161:13-17.
17. Goldsteins G, Persson H, Andersson K, et al. Exposure of cryptic epitopes on transthyretin only in amyloid and in amyloidogenic mutants. Proc Natl Acad Sci USA 1999;96:3108-3113.
18. Redondo C, Damas AM, Olofsson A, et al. Search for intermediate structures in transthyretin fibrillogenesis: soluble tetrameric Tyr78Phe TTR expresses a specific epitope present only in amyloid fibrils. J Mol Biol 2000;304:461-470.
19. Hock C, Konietzko U, Papassotiropoulos A, et al. Generation of antibodies specific for β-amyloid by vaccination of patients with Alzheimer disease. Nat Med 2002;8:1270-1275.
20. Kohno K, Palha JA, Miyakawa K, et al. Analysis of amyloid deposition in a transgenic mouse model of homozygous familial amyloidotic polyneuropathy. Am J Pathol 1997;150:1497-1508.
21. Sousa MM, Fernandes R, Palha JA, et al. Evidence for early cytotoxic aggregates in transgenic mice for human transthyretin Leu55Pro. Am J Pathol 2002;161:1935-1948.
22. Episkopou V, Maeda S, Nishiguchi S, et al. Disruption of the transthyretin gene results in mice with depressed levels of plasma retinol and thyroid hormone. Proc Natl Acad Sci USA 1993;90:2375-2379.
23. Puchtler H, Sweat F. Congo red as a stain for fluorescence microscopy of amyloid. J Histochem Cytochem 1965;13:693-694.
24. Monsonego A, Maron R, Zota V, et al. Immune hyporesponsiveness to amyloid β-peptide in amyloid precursor protein transgenic mice: implications for the pathogenesis and treatment of Alzheimer's disease. Proc Nat Acad Sci USA 2001;98:10273-10278.
25. Sousa MM, Cardoso I, Fernandes R, et al. Deposition of transthyretin in early stages of familial amyloidotic polyneuropathy: evidence for toxicity of nonfibrillar aggregates. Am J Pathol 2001;159:1993-2000.
26. Das P, Howard V, Loosbrock N, et al. Amyloid-β immunization effectively reduced amyloid deposition in FcRγ-/- knock-out mice. J Neurosci 2003;23:8532-8538.
27. McLaurin J, Cecal R, Kierstead ME, et al. Therapeutically effective antibodies against amyloid-β peptide target amyloid-β residues 4-10 and inhibit cytotoxicity and fibrillogenesis. Nat Med 2002;8:1263-1269.
28. Schenk D. Amyloid β immunotherapy for Alzheimer's disease: the end of the beginning. Nat Rev Neurosci 2002;3:824-828.
29. Senior K. Dosing in phase II trial of Alzheimer's vaccine suspended. Lancet Neurol 2002;1:3.