A novel mechanism of complement-independent clearance of red cells deficient in glycosyl phosphatidylinositol-linked proteins

Blood

Volumn 103, Issue 7, 2004, Pages 2827-2834

  Jasinski, Marek ^b   Pantazopoulos, Panagiotis ^b   Rother, Russell P ^b   Van Rooijen, Nico ^b   Song, Wen Chao ^b   Molina, Hector ^b   Bessler, Monica ^a  

^a University School of Medicine   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

ALTERNATIVE COMPLEMENT PATHWAY C3 C5 CONVERTASE; CLODRONIC ACID; COMPLEMENT; COMPLEMENT COMPONENT C3; COMPLEMENT COMPONENT C4; COMPLEMENT COMPONENT C5; CORTISONE; GLYCOSYLPHOSPHATIDYLINOSITOL; GLYCOSYLPHOSPHATIDYLINOSITOL LINKED PROTEIN; LIPOSOME; PROTEIN; UNCLASSIFIED DRUG;

ACQUIRED HEMOLYTIC ANEMIA; ANIMAL CELL; ARTICLE; CELL CLONE; CELL SURVIVAL; COMPLEMENT SYSTEM; CONTROLLED STUDY; DRUG MEGADOSE; ERYTHROCYTE; GENE; GENE INACTIVATION; GENE MUTATION; GENE TARGETING; HALF LIFE TIME; HEMOGLOBINURIA; INTRAVASCULAR HEMOLYSIS; MACROPHAGE; MOUSE; NONHUMAN; PAROXYSMAL NOCTURNAL HEMOGLOBINURIA; PIGA GENE; PRIORITY JOURNAL; SPLENECTOMY;

EID: 1642301159     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2003-09-3057     Document Type: Article

References (39)

1. Ham TH. Chronic hemolytic anemia with paroxysmal nocturnal hemoglobinuria: study of the mechanism of hemolysis in relation to acid-base equilibrium. N Engl J Med. 1937;217:915-918.
2. Takeda J, Miyata T, Kawagoe K, et al. Deficiency of the GPI anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria. Cell. 1993;73:703-711.
3. Nicholson-Weller A, March JP, Rosenfeld SI, Austen KF. Affected erythrocytes of patients with paroxysmal nocturnal hemoglobinuria are deficient in the complement regulatory protein, decay accelerating factor. Proc Natl Acad Sci U S A. 1983;80:5066-5070.
4. Holguin MH, Fredrick LR, Bernshaw NJ, Wilcox LA, Parker CJ. Isolation and characterization of a membrane protein from normal human erythrocytes that inhibits reactive lysis of the erythrocytes of paroxysmal nocturnal hemoglobinuria. J Clin Invest. 1989;84:7-17.
5. Tremml G, Dominguez C, Rosti V, et al. Increased sensitivity to complement and a decreased red blood cell life span in mice mosaic for a nonfunctional Piga gene. Blood. 1999;94:2945-2954.
6. Keller P, Payne JL, Tremml G, et al. FES-Cre targets PIGA inactivation to hematopoietic stem cells in the bone marrow. J Exp Med. 2001;194:581-589.
7. Jasinski M, Keller P, Fujiwara Y, Orkin SH, Bessler M. GATA1-Cre mediates Piga gene inactivation in the erythroid/megakaryocytic lineage and leads to circulating red cells with a partial deficiency in glycosyl phosphatidylinositol-linked proteins (paroxysmal nocturnal hemoglobinuria type II cells). Blood. 2001;98:2248-2255.
8. Powell MB, Marchbank KJ, Rushmere NK, van den Berg CW, Morgan BP. Molecular cloning, chromosomal localization, expression, and functional characterization of the mouse analogue of human CD59. J Immunol. 1997;158:1692-1702.
9. Qian YM, Qin X, Miwa T, Sun X, Halperin JA, Song WC. Identification and functional characterization of a new gene encoding the mouse terminal complement inhibitor CD59. J Immunol. 2000;165:2528-2534.
10. Qin X, Miwa T, Aktas H, et al. Genomic structure, functional comparison, and tissue distribution of mouse Cd59a and Cd59b. Mamm Genome. 2001;12:582-589.
11. Harris CL, Hanna SM, Mizuno M, Holt DS, Marchbank KJ, Morgan BP. Characterization of the mouse analogues of CD59 using novel monoclonal antibodies: tissue distribution and functional comparison. Immunology. 2003;109:117-126.
12. Spicer AP, Seldin MF, Gendler SJ. Molecular cloning and chromosomal localization of the mouse decay-accelerating factor genes: duplicated genes encode glycosylphosphatidylinositol-anchored and transmembrane forms. J Immunol. 1995;155:3079-3091.
13. Holers VM, Kinoshita T, Molina H. The evolution of mouse and human complement C3-binding proteins: divergence of form but conservation of function. Immunol Today. 1992;13:231-236.
14. Wessels MR, Butko P, Ma M, Warren HB, Lage AL, Carroll MC. Studies of group B streptococcal infection in mice deficient in complement component C3 or C4 demonstrate an essential role for complement in both innate and acquired immunity. Proc Natl Acad Sci U S A. 1995;92:11490-11494.
15. Matsumoto M, Fukuda W, Circolo A, et al. Abrogation of the alternative complement pathway by targeted deletion of murine factor B. Proc Natl Acad Sci U S A. 1997;94:8720-8725.
16. Circolo A, Garnier G, Fukuda W, et al. Genetic disruption of the murine complement C3 promoter region generates deficient mice with extrahepatic expression of C3 mRNA. Immunopharmacology. 1999;42:135-149.
17. Sun X, Funk CD, Deng C, Sahu A, Lambris JD, Song WC. Role of decay-accelerating factor in regulating complement activation on the erythrocyte surface as revealed by gene targeting. Proc Natl Acad Sci U S A. 1999;96:628-633.
18. Miwa T, Zhou L, Hilliard B, Molina H, Song WC. Crry, but not CD59 and DAF, is indispensable for murine erythrocyte protection in vivo from spontaneous complement attack. Blood. 2002;99:3707-3716.
19. Berden JH, Hagemann JF, Koene RA. A sensitive haemolytic assay of mouse complement. J Immunol Methods. 1978;23:149-159.
20. Gorman JC, Jackson R, Desantola JR, Shreffler D, Atkinson JP. Development of a hemolytic assay for mouse C2 and determination of its genetic control. J Immunol. 1980;125:344-351.
21. Atkinson JP, McGinnis K, Shreffler D. Development and characterization of a hemolytic assay for mouse C4. J Immunol Methods. 1980;33:351-368.
22. Holt DS, Botto M, Bygrave AE, Hanna SM, Walport MJ, Morgan BP. Targeted deletion of the CD59 gene causes spontaneous intravascular hemolysis and hemoglobinuria. Blood. 2001;98:442-449.
23. Molina H, Miwa T, Zhou L, et al. Complement-mediated clearance of erythrocytes: mechanism and delineation of the regulatory roles of Crry and DAF. Blood. 2002;100:4544-4549.
24. Wang Y, Rollins SA, Madri JA, Matis LA. Anti-C5 monoclonal antibody therapy prevents collagen-induced arthritis and ameliorates established disease. Proc Natl Acad Sci U S A. 1995;92:8955-8959.
25. Ganick DJ, Segel GB, Chamberlain J, Hirsch L, Klemperer MR. The effects of splenectomy and glucocorticoids on survival and hepatic uptake of damaged red cells in the mouse. Am J Hematol. 1977;2:365-373.
26. van Rooijen N, van Nieuwmegen R, Kamperdijk EW. Elimination of phagocytic cells in the spleen after intravenous injection of liposome-encapsulated dichloromethylene diphosphonate: ultrastructural aspects of elimination of marginal zone macrophages. Virchows Arch B Cell Pathol Incl Mol Pathol. 1985;49:375-383.
27. Jordan MB, van Rooijen N, Izui S, Kappler J, Marrack P. Liposomal clodronate as a novel agent for treating autoimmune hemolytic anemia in a mouse model. Blood. 2003;101:594-601.
28. Van Rooijen N, Sanders A. Liposome mediated depletion of macrophages: mechanism of action, preparation of liposomes and applications. J Immunol Methods. 1994;174:83-93.
29. Atkinson JP, McGinnis K, Brown L, Peterein J, Shreffler D. A murine C4 molecule with reduced hemolytic efficiency. J Exp Med. 1980;151:492-497.
30. Ebanks RO, Isenman DE. Mouse complement component C4 is devoid of classical pathway C5 convertase subunit activity. Mol Immunol. 1996;33:297-309.
31. Van den Berg CW, Aerts PC, Van Dijk H. In vivo anti-complementary activities of the cobra venom factors from Naja naja and Naja haje. J Immunol Methods. 1991;136:287-294.
32. Rosse WF. The life-span of complement-sensitive and -insensitive red cells in paroxysmal nocturnal hemoglobinuria. Blood. 1971;37:556-562.
33. Yonemura Y, Kawakita M, Koito A, et al. Paroxysmal nocturnal hemoglobinuria with coexisting deficiency of the ninth component of complement: lack of massive haemolytic attack. Brit J Haematol. 1990;74:108-113.
34. Hillmen P, Hall C, Marsh J, et al. Eculizumab, a C5 complement-blocking antibody, abolishes hemolysis and renders hemolytic patients with paroxysmal nocturnal hemoglobinuria (PNH) transfusion independent [abstract]. Blood. 2002;100;44a.
35. Qin X, Krumrei N, Grubissich L, et al. Deficiency of the mouse complement regulatory protein mCd59b results in spontaneous hemolytic anemia with platelet activation and progressive male infertility. Immunity. 2003;18:217-227.
36. Li B, Sallee C, Dehoff M, Foley S, Molina H, Holers VM. Mouse Crry/p65: characterization of monoclonal antibodies and the tissue distribution of a functional homologue of human MCP and DAF. J Immunol. 1993;151:4295-4305.
37. Telen MJ, Hall SE, Green AM, Moulds JJ, Rosse WF. Identification of human erythrocytes blood group antigens on decay accelerating factor (DAF) and on erythrocyte phenotype negative for DAF. J Exp Med. 1988;167:1993-1998.
38. Merry AH, Rawlinson VI, Uchikawa M, Daha MR, Sim RB. Studies on the sensitivity to complement-mediated lysis of erythrocytes (Inab phenotype) with a deficiency of DAF (decay accelerating factor). Brit J Haematol. 1989;73:248-253.
39. Holguin MH, Martin CB, Bernshaw NJ, Parker CJ. Analysis of the effects of activation of the alternative pathway of complement on erythrocytes with an isolated deficiency of decay accelerating factor. J Immunol. 1992;148:498-502.