Mouse models of sickle cell disease

Transfusion Clinique et Biologique

Volumn 15, Issue 1-2, 2008, Pages 7-11

  Beuzard, Y ^a,b,c  

^a INSERM   (France)

^b DIF   (France)

^c UNIVERSITÉ PARIS DESCARTES   (France)

Author keywords

Gene therapy; Mouse model; Sickle cell anemia; Stem cells

Indexed keywords

HEMOGLOBIN F; HEMOGLOBIN S;

ALPHA THALASSEMIA; ARTICLE; BETA THALASSEMIA; DISEASE MODEL; EMBRYONIC STEM CELL; GENE THERAPY; GLOBIN GENE; KNOCKOUT MOUSE; NONHUMAN; PATHOPHYSIOLOGY; SICKLE CELL; TRANSGENIC MOUSE;

ANEMIA, SICKLE CELL; ANIMALS; DISEASE MODELS, ANIMAL; GENE THERAPY; HEMOGLOBIN, SICKLE; MICE; MICE, KNOCKOUT; MICE, TRANSGENIC;

EID: 45049085401     PISSN: 12467820     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.tracli.2008.04.001     Document Type: Article

References (39)

1. Grosveld F., van Assendelft G.B., Greaves D.R., and Kollias G. Position-independent, high-level expression of the human beta-globin gene in transgenic mice. Cell 51 6 (1987) 975-985
2. Behringer R.R., Ryan T.M., Reilly M.P., Asakura T., Palmiter R.D., Brinster R.L., et al. Synthesis of functional human hemoglobin in transgenic mice. Science 245 4921 (1989) 971-973
3. Rhoda M.D., Domenget C., Vidaud M., Bardakdjian-Michau J., Rouyer-Fessard P., Rosa J., et al. Mouse alpha chains inhibit polymerization of hemoglobin induced by human beta S or beta S Antilles chains. Biochim Biophys Acta 952 2 (1988) 208-212
4. Greaves D.R., Fraser P., Vidal M.A., Hedges M.J., Ropers D., Luzzatto L., et al. A transgenic mouse model of sickle cell disorder. Nature 343 6254 (1990) 183-185
5. Ryan T.M., Townes T.M., Reilly M.P., Asakura T., Palmiter R.D., Brinster R.L., et al. Human sickle hemoglobin in transgenic mice. Science 247 4942 (1990) 566-568
6. Trudel M., Saadane N., Garel M.C., Bardakdjian-Michau J., Blouquit Y., Guerquin-Kern J.L., et al. Towards a transgenic mouse model of sickle cell disease: hemoglobin SAD. Embo J 10 11 (1991) 3157-3165
7. Trudel M., De Paepe M.E., Chrétien N., Saadane N., Jacmain J., Sorette M., et al. Sickle cell disease of transgenic SAD mice. Blood 84 9 (1994) 3189-3197
8. De Franceschi L., Beuzard Y., Jouault H., and Brugnara C. Modulation of erythrocyte potassium chloride cotransport, potassium content and density by dietary magnesium intake in transgenic SAD mouse. Blood 88 7 (1996) 2738-2744
9. De Franceschi L., Saadane N., Trudel M., Alper S.L., Brugnara C., and Beuzard Y. Treatment with oral clotrimazole blocks Ca(2+)-activated K+ transport and reverses erythrocyte dehydration in transgenic SAD mice. A model for therapy of sickle cell disease. J Clin Invest 93 4 (1994) 1670-1676
10. De Paepe M.E., and Trudel M. The transgenic SAD mouse: a model of human sickle cell glomerulopathy. Kidney Int 46 5 (1994) 1337-1345
11. De Franceschi L., Brugnara C., Rouyer-Fessard P., Jouault H., and Beuzard Y. Formation of dense erythrocytes in SAD mice exposed to chronic hypoxia: evaluation of different therapeutic regimens and of a combination of oral clotrimazole and magnesium therapies. Blood 94 12 (1999) 4307-4313
12. De Franceschi L., Bachir D., Galacteros F., Tchernia G., Cynober T., Alper S., et al. Oral magnesium supplements reduce erythrocyte dehydration in patients with sickle cell disease. J Clin Invest 100 7 (1997) 1847-1852
13. De Franceschi L., Bachir D., Galacteros F., Tchernia G., Cynober T., Neuberg D., et al. Oral magnesium pidolate: effects of long-term administration in patients with sickle cell disease. Br J Haematol 108 2 (2000) 284-289
14. Hankins J.S., Wynn L.W., Brugnara C., Hillery C.A., Li C.S., and Wang W.C. Phase I study of magnesium pidolate in combination with hydroxycarbamide for children with sickle cell anaemia. Br J Haematol 140 1 (2008) 80-85
15. Stocker J.W., De Franceschi L., McNaughton-Smith G.A., Corrocher R., Beuzard Y., and Brugnara C. ICA-17043, a novel Gardos channel blocker, prevents sickled red blood cell dehydration in vitro and in vivo in SAD mice. Blood 101 6 (2003) 2412-2418
16. Ataga K.I., Smith W.R., De Castro L.M., Swerdlow P., Saunthararajah Y., Castro O., et al. Efficacy and safety of the Gardos channel blocker, Senicapoc (ICA-17043), in patients with sickle cell anemia. Blood (2008)
17. Gill F.M., Sleeper L.A., Weiner S.J., Brown A.K., Bellevue R., Grover R., et al. Clinical events in the first decade in a cohort of infants with sickle cell disease. Cooperative Study of Sickle Cell Disease. Blood 86 2 (1995) 776-783
18. de Franceschi L., Baron A., Scarpa A., Adrie C., Janin A., Barbi S., et al. Inhaled nitric oxide protects transgenic SAD mice from sickle cell disease-specific lung injury induced by hypoxia/reoxygenation. Blood 102 3 (2003) 1087-1096
19. de Franceschi L., Malpeli G., Scarpa A., Janin A., Muchitsch E.M., Roncada P., et al. Protective effects of S-nitrosoalbumin on lung injury induced by hypoxia-reoxygenation in mouse model of sickle cell disease. Am J Physiol Lung Cell Mol Physiol 291 3 (2006) L457-L465
20. De Franceschi L., Platt O.S., Malpeli G., Janin A., Scarpa A., Leboeuf C., et al. Protective effects of phosphodiesterase-4 (PDE-4) inhibition in the early phase of pulmonary arterial hypertension in transgenic sickle cell mice. Faseb J (2008)
21. Bonnin P., Sabaa N., Flamant M., Debbabi H., and Tharaux P.L. Ultrasound imaging of renal vaso-occlusive events in transgenic sickle mice exposed to hypoxic Stress. Ultrasound Med Biol (2008)
22. Fabry M.E., Sengupta A., Suzuka S.M., Costantini F., Rubin E.M., Hofrichter J., et al. A second generation transgenic mouse model expressing both hemoglobin S (HbS) and HbS-Antilles results in increased phenotypic severity. Blood 86 6 (1995) 2419-2428
23. Nath K.A., Grande J.P., Haggard J.J., Croatt A.J., Katusic Z.S., Solovey A., et al. Oxidative stress and induction of heme oxygenase-1 in the kidney in sickle cell disease. Am J Pathol 158 3 (2001) 893-903
24. Belcher J.D., Mahaseth H., Welch T.E., Otterbein L.E., Hebbel R.P., and Vercellotti G.M. Heme oxygenase-1 is a modulator of inflammation and vaso-occlusion in transgenic sickle mice. J Clin Invest 116 3 (2006) 808-816
25. Kennan R.P., Suzuka S.M., Nagel R.L., and Fabry M.E. Decreased cerebral perfusion correlates with increased BOLD hyperoxia response in transgenic mouse models of sickle cell disease. Magn Reson Med 51 3 (2004) 525-532
26. Fasipe F.R., Ubawike A.E., Eva R., and Fabry M.E. Arginine supplementation improves rotorod performance in sickle transgenic mice. Hematology 9 4 (2004) 301-305
27. Ryan T.M., Ciavatta D.J., and Townes T.M. Knockout-transgenic mouse model of sickle cell disease. Science 278 5339 (1997) 873-876
28. Paszty C., Brion C.M., Manci E., Witkowska H.E., Stevens M.E., Mohandas N., et al. Transgenic knockout mice with exclusively human sickle hemoglobin and sickle cell disease. Science 278 5339 (1997) 876-878
29. Fabry M.E., Suzuka S.M., Weinberg R.S., Lawrence C., Factor S.M., Gilman J.G., et al. Second generation knockout sickle mice: the effect of HbF. Blood 97 2 (2001) 410-418
30. Chang J.C., Lu R., Lin C., Xu S.M., Kan Y.W., Porcu S., et al. Transgenic knockout mice exclusively expressing human hemoglobin S after transfer of a 240-kb betas-globin yeast artificial chromosome: a mouse model of sickle cell anemia. Proc Natl Acad Sci U S A 95 25 (1998) 14886-14890
31. Wu L.C., Sun C.W., Ryan T.M., Pawlik K.M., Ren J., and Townes T.M. Correction of sickle cell disease by homologous recombination in embryonic stem cells. Blood 108 4 (2006) 1183-1188
32. Hanna J., Wernig M., Markoulaki S., Sun C.W., Meissner A., Cassady J.P., et al. Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin. Science 318 5858 (2007) 1920-1923
33. Wood K.C., Hsu L.L., and Gladwin M.T. Sickle cell disease vasculopathy: a state of nitric oxide resistance. Free Radic Biol Med (2008)
34. Hsu L.L., Champion H.C., Campbell-Lee S.A., Bivalacqua T.J., Manci E.A., Diwan B.A., et al. Hemolysis in sickle cell mice causes pulmonary hypertension due to global impairment in nitric oxide bioavailability. Blood 109 7 (2007) 3088-3098
35. Iannone R., Luznik L., Engstrom L.W., Tennessee S.L., Askin F.B., Casella J.F., et al. Effects of mixed hematopoietic chimerism in a mouse model of bone marrow transplantation for sickle cell anemia. Blood 97 12 (2001) 3960-3965
36. Levasseur D.N., Ryan T.M., Reilly M.P., McCune S.L., Asakura T., and Townes T.M. A recombinant human hemoglobin with anti-sickling properties greater than fetal hemoglobin. J Biol Chem 279 26 (2004) 27518-27524
37. Hanawa H., Hargrove P.W., Kepes S., Srivastava D.K., Nienhuis A.W., and Persons D.A. Extended beta-globin locus control region elements promote consistent therapeutic expression of a gamma-globin lentiviral vector in murine beta-thalassemia. Blood 104 8 (2004) 2281-2290
38. Samakoglu S., Lisowski L., Budak-Alpdogan T., Usachenko Y., Acuto S., Di Marzo R., et al. A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference. Nat Biotechnol 24 1 (2006) 89-94
39. Saaba N., de Franceschi L., Bonnin P., Castier Y., Malpeli G., Debbabi H., et al. Endothelin receptor antagonism prevents hypoxia-induced mortality and morbidity in a mouse model of sickle-cell disease. J Clin Invest 118 5 (2008) 1924-1933