Transferrin Fusion Protein Therapies: Acetylcholine Receptor-Transferrin Fusion Protein as a Model

Fusion Protein Technologies for Biopharmaceuticals: Applications and Challenges

Volumn , Issue , 2013, Pages 191-199

  Keefe, Dennis ^a   Heartlein, Michael ^a   Josiah, Serene ^a  

^a Shire Human Genetic Therapies   (United States)

Author keywords

Acetylcholine, transferrin; Antigenic modulation; FP SHG2210 design; SHG2210 characterization; Transferrin FP

Indexed keywords

EID: 84886105614     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9781118354599.ch12     Document Type: Chapter

References (49)

1. Conti-Fine BM, et al. (2006) Myasthenia gravis: past, present, and future. J. Clin. Invest. 116(11), 2843-2854.
2. Biesecker G, Gomez CM. (1989) Inhibition of acute passive transfer experimental autoimmune myasthenia gravis with Fab antibody to complement C6. J. Immunol. 142(8), 2654-2659.
3. Lennon VA, et al. (1978) Role of complement in the pathogenesis of experimental autoimmune myasthenia gravis. J. Exp. Med. 147(4), 973-983.
4. Sahashi K, et al. (1980) Ultrastructural localization of the terminal and lytic ninth complement component (C9) at the motor end-plate in myasthenia gravis. J. Neuropathol. Exp. Neurol. 39(2), 160-172.
5. Hoch W, et al. (2001) Auto-antibodies to the receptor tyrosine kinase MuSK in patients with myasthenia gravis without acetylcholine receptor antibodies. Nat. Med. 7(3), 365-368.
6. Newsom-Davis J, et al. (1978) Function of circulating antibody to acetylcholine receptor in myasthenia gravis: investigation by plasma exchange. Neurology 28(3), 266-272.
7. Christadoss P, et al. (2000) Animal models of myasthenia gravis. Clin. Immunol. 94(2), 75-87.
8. Tzartos S, et al. (1987) Passive transfer of experimental autoimmune myasthenia gravis by monoclonal antibodies to the main immunogenic region of the acetylcholine receptor. J. Neuroimmunol. 15(2), 185-194.
9. Garcia-Carrasco M, et al. (2007) Therapeutic options in autoimmune myasthenia gravis. Autoimmun. Rev. 6(6), 373-378.
10. Kalamida D, et al. (2007) Muscle and neuronal nicotinic acetylcholine receptors. Structure, function and pathogenicity. FEBS J. 274(15), 3799-3845.
11. Kostelidou K, et al. (2007) Extracellular domains of the beta, gamma and epsilon subunits of the human acetylcholine receptor as immunoadsorbents for myasthenic autoantibodies: a combination of immunoadsorbents results in increased efficiency. J. Neuroimmunol. 190(1,2), 44-52.
12. Loutrari H, et al. (1997) Expression of human-Torpedo hybrid acetylcholine receptor (AChR) for analysing the subunit specificity of antibodies in sera from patients with myasthenia gravis (MG). Clin. Exp. Immunol. 109(3), 538-546.
13. Sideris S, et al. (2007) Isolation and functional characterization of anti-acetylcholine receptor subunit-specific autoantibodies from myasthenic patients: receptor loss in cell culture. J. Neuroimmunol. 189(1,2), 111-117.
14. Barchan D, et al. (1998) Modulation of the anti-acetylcholine receptor response and experimental autoimmune myasthenia gravis by recombinant fragments of the acetylcholine receptor. Eur. J. Immunol. 28(2), 616-624.
15. Bleil JD, Bretscher MS. (1982) Transferrin receptor and its recycling in HeLa cells. EMBO J. 1(3), 351-355.
16. Lamb JE, et al. (1983) Internalization and subcellular localization of transferrin and transferrin receptors in HeLa cells. J. Biol. Chem. 258(14), 8751-8758.
17. Sager PR, et al. (1984) Analysis of transferrin recycling in mitotic and interphase HeLa cells by quantitative fluorescence microscopy. Cell 39(2 Pt 1), 275-282.
18. Hradilek A, Neuwirt J. (1986) Iron uptake by MOLT 3 cells from transferrin/monoclonal antitransferrin antibody complexes. Br. J. Haematol. 62(1), 21-30.
19. Keefe D, et al. In vitro characterization of an acetylcholine receptor-transferrin fusion protein for the treatment of myasthenia gravis. Autoimmunity 43(8), 628-639.
20. Tzartos SJ, et al. (1988) Localization of the main immunogenic region of human muscle acetylcholine receptor to residues 67-76 of the alpha subunit. Proc. Natl. Acad. Sci. USA 85(9), 2899-2903.
21. Tsouloufis T, et al. (2000) Reconstitution of conformationally dependent epitopes on the N-terminal extracellular domain of the human muscle acetylcholine receptor alpha subunit expressed in Escherichia coli: implications for myasthenia gravis therapeutic approaches. Int. Immunol. 12(9), 1255-1265.
22. Beeson D, et al. (1996) A transfected human muscle cell line expressing the adult subtype of the human muscle acetylcholine receptor for diagnostic assays in myasthenia gravis. Neurology 47(6), 1552-1555.
23. Bruneau E, et al. (2005) Identification of nicotinic acetylcholine receptor recycling and its role in maintaining receptor density at the neuromuscular junction in vivo. J. Neurosci. 25(43), 9949-9959.
24. Bruneau EG, Akaaboune M. (2006) The dynamics of recycled acetylcholine receptors at the neuromuscular junction in vivo. Development 133(22), 4485-4493.
25. Kumari S, et al. (2008) Nicotinic acetylcholine receptor is internalized via a Rac-dependent, dynamin-independent endocytic pathway. J. Cell. Biol. 181(7), 1179-1193.
26. Heinemann S, et al. (1977) Modulation of acetylcholine receptor by antibody against the receptor. Proc. Natl. Acad. Sci. USA 74(7), 3090-3094.
27. Lindstrom J, Einarson B. (1979) Antigenic modulation and receptor loss in experimental autoimmune myasthenia gravis. Muscle Nerve 2(3), 173-179.
28. Reiness CG, et al. (1978) Antibody to acetylcholine receptor increases degradation of junctional and extrajunctional receptors in adult muscle. Nature 274(5666), 68-70.
29. Keefe D, et al. (2009) A rapid, fluorescence-based assay for detecting antigenic modulation of the acetylcholine receptor on human cell lines. Cytometry B. Clin. Cytom. 76(3), 206-212.
30. Barchan D, et al. (1999) Antigen-specific modulation of experimental myasthenia gravis: nasal tolerization with recombinant fragments of the human acetylcholine receptor alphasubunit. Proc. Natl. Acad. Sci. USA 96(14), 8086-8091.
31. Hossann M, et al. (2006) Novel immunotoxin: a fusion protein consisting of gelonin and an acetylcholine receptor fragment as a potential immunotherapeutic agent for the treatment of Myasthenia gravis. Protein Expr. Purif. 46(1), 73-84.
32. Chang T, et al. Selective recognition and elimination of nicotinic acetylcholine receptor-reactive B cells by a recombinant fusion protein AChR-Fc in myasthenia gravis in vitro. J. Neuroimmunol. 227(1,2), 35-43.
33. Parise F, et al. (1999) Construction and in vitro functional evaluation of a low-density lipoprotein receptor/transferrin fusion protein as a therapeutic tool for familial hypercholesterolemia. Hum. Gene. Ther. 10(7), 1219-1228.
34. Moreland LW, et al. (1997) Treatment of rheumatoid arthritis with a recombinant human tumor necrosis factor receptor (p75)-Fc fusion protein. N. Engl. J. Med. 337(3), 141-147.
35. Moreland LW, et al. (1997) Biologic agents for treating rheumatoid arthritis. Concepts and progress. Arthritis Rheum. 40(3), 397-409.
36. Marsh DR, et al. (2002) Neutralizing intraspinal nerve growth factor with a trkA-IgG fusion protein blocks the development of autonomic dysreflexia in a clip-compression model of spinal cord injury. J. Neurotrauma. 19(12), 1531-1541.
37. Meyuhas R, et al. (2009) Enhanced HIV-1 neutralization by a CD4-VH3-IgG1 fusion protein. Biochem. Biophys. Res. Commun. 386(2), 402-406.
38. Schapira K, et al. (2009) Fn14-Fc fusion protein regulates atherosclerosis in ApoE-/- mice and inhibits macrophage lipid uptake in vitro. Arterioscler. Thromb. Vasc. Biol. 29(12), 2021-2027.
39. Qian ZM, et al. (2002) Targeted drug delivery via the transferrin receptor-mediated endocytosis pathway. Pharmacol. Rev. 54(4), 561-587.
40. Fritzer M, et al. (1996) Cytotoxic effects of a doxorubicintransferrin conjugate in multidrug-resistant KB cells. Biochem. Pharmacol. 51(4), 489-493.
41. Lai BT, et al. (1998) Mechanism of action and spectrum of cell lines sensitive to a doxorubicin-transferrin conjugate. Cancer Chemother. Pharmacol. 41(2), 155-160.
42. Singh M, et al. (1998) Transferrin directed delivery of adriamycin to human cells. Anticancer Res. 18(3A) 1423-1427.
43. Laske DW, et al. (1994) Efficacy of direct intratumoral therapy with targeted protein toxins for solid human gliomas in nude mice. J. Neurosurg. 80(3), 520-526.
44. Bickel U, et al. (1993) Pharmacologic effects in vivo in brain by vector-mediated peptide drug delivery. Proc. Natl. Acad. Sci. USA 90(7), 2618-2622.
45. Pardridge WM, et al. (1998) Combined use of carboxyldirected protein pegylation and vector-mediated blood-brain barrier drug delivery system optimizes brain uptake of brainderived neurotrophic factor following intravenous administration. Pharm. Res. 15(4), 576-582.
46. Cheng PW. (1996) Receptor ligand-facilitated gene transfer: enhancement of liposome-mediated gene transfer and expression by transferrin. Hum. Gene Ther. 7(3), 275-282.
47. Simoes S, et al. (1999) Cationic liposomes as gene transfer vectors: barriers to successful application in gene therapy. Curr. Opin. Mol. Ther. 1(2), 147-157.
48. Simoes S, et al. (1998) Gene delivery by negatively charged ternary complexes of DNA, cationic liposomes and transferrin or fusigenic peptides. Gene Ther. 5(7), 955-964.
49. Psaridi-Linardaki L, et al. (2005) Specific immunoadsorption of the autoantibodies from myasthenic patients using the extracellular domain of the human muscle acetylcholine receptor alpha-subunit. Development of an antigen-specific therapeutic strategy. J. Neuroimmunol. 159(1,2), 183-191.