Mechanisms leading to sustained reversion of β-thalassemia in mice by doxycycline-controlled epo delivery from muscles

Molecular Therapy

Volumn 6, Issue 6, 2002, Pages 793-803

  Samakoglu, Selda ^a   Bohl, Delphine ^a   Heard, Jean Michel ^a  

^a INSTITUT PASTEUR   (France)

Author keywords

AAV vectors; Epo; Erythroid progenitors; Erythropoiesis; Fetal hemoglobin; thalassemia

Indexed keywords

ADENOVIRUS VECTOR; BUTYRIC ACID DERIVATIVE; DOXYCYCLINE; GLOBIN; HEMOGLOBIN; HUMAN IMMUNOGLOBULIN; HYDROXYUREA; RECOMBINANT ERYTHROPOIETIN; TETRACYCLINE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; BETA THALASSEMIA; BURST FORMING UNIT E; CELL MATURATION; CIRCULATION; CONTROLLED STUDY; ERYTHROCYTE; ERYTHROID PRECURSOR CELL; ERYTHROPOIESIS; FOLLOW UP; GENE THERAPY; GENETIC ENGINEERING; GLOBIN SYNTHESIS; HEMATOCRIT; HEMOGLOBIN SYNTHESIS; HUMAN; MOUSE; NONHUMAN; REVIEW; SKELETAL MUSCLE; STEADY STATE; STRESS; VIRAL GENE DELIVERY SYSTEM;

ADENOVIRIDAE; ANIMALIA;

EID: 0036932835     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2002.0810     Document Type: Review

References (54)

1. Aljurf, M., et al. (1996). Abnormal assembly of membrane proteins in erythroid progenitors of patients with β-thalassemia major. Blood 87: 2049-2056.
2. Shinar, E., and Rachmilewitz, E. (1990). Oxidative denaturation of red blood cells in thalassemia. Semin. Hematol. 27: 70-82.
3. Mathias, L. A., et al. (2000). Ineffective erythropoiesis in β-thalassemia major is due to apoptosis at the polychromatophilic normoblast stage. Exp. Hematol. 28: 1343-1353.
4. Manor, D., Fibach, E., Goldfarb, A., and Rachmilevitch, E. A. (1986). Erythropoietin activity in the serum of β-thalassemic patients. Scand. J. Haematol. 37: 221-228.
5. Galanello, R., et al. (1994): Serum erythropoietin and erythropoiesis in high- and low-fetal hemoglobin β-thalassemia intermedia patients. Blood 83: 561-565.
6. Olivieri, N. F., et al. (1997). Treatment of thalassemia major with phenylbutyrate and hydroxyurea. Lancet 350: 491-492.
7. Umemura, T., Al Khatti, A., Donahue, R. E., Papayannopoulou, T., and Stamatoyanno-poulos, G. (1989). Effects of interleukin-3 and erythropoietin on in vivo erythropoiesis and F-cell formation in primates. Blood 74: 1571-1576.
8. Kauchansky, K., et al. (1995). Thrombopoietin expands erythroid progenitors, increases red cell production, and enhances erythroid recovery after myelosuppressive therapy. J. Clin. Invest. 96: 1683-1687.
9. Gabbianelli, M., et al. (2000). Hemoglobin switching in unicellular erythroid culture of sibling erythroid burst-forming units: Kit ligand induces a dose-dependent fetal hemoglobin reactivation potentiated by sodium butyrate. Blood 95: 3555-3561.
10. Alter, B. P., Rappeport, J. M., Huisman, T. H., Schroeder, W. A., and Nathan, D. G. (1976). Fetal erythropoiesis following bone marrow transplantation. Blood 48: 843-853.
11. Papayanopoulou, T., Vichinsky, E., and Stamatoyannopoulos, G. (1980). Fetal Hb production during acute erythroid expansion. I. Observations in patients with transient erythroblastopenia and post-phlebotomy. Br. J. Haematol. 44: 535-546.
12. Link, M. P., and Alter, B. P. (1981). Fetal-like erythropoiesis during recovery from transient erythroblastopenia of childhood (TEC). Pediatr. Res. 15: 1036-1039.
13. Wood, W., Stamatoyannopoulos, G., and Nute, P. (1975). F-cells in the adult: Normal values and levels in individuals with hereditary and acquired elevations of Hb F. Blood 46: 671-682.
14. Zago, M. A., et al. (1979). Genetic control of F cells in human adults. Blood 53: 977-986.
15. Papayannopoulou, T., Kalmantis, T., and Stamatoyannopoulos, G. (1979). Cellular regulation of hemoglobin switching: Evidence for inverse relationship between fetal hemoglobin synthesis and degree of maturity of human erythroid cells. Proc. Natl. Acad. Sci. USA 76: 6420-6424.
16. Umemura, T., Al-Khatti, A., Papayannopoulou, T., and Stamatoyannopoulos, G. (1988). Fetal hemoglobin synthesis in vivo: Direct evidence for control at the level of erythroid progenitors. Proc. Natl. Acad. Sci. USA 85: 9278-9282.
17. Stamatoyannopoulos, G., Kurnit, D., and Papayannopoulou, T. (1981). Stochastic expression of fetal hemoglobin in adult erythroid cells. Proc. Natl. Acad. Sci. USA 78: 7005-7009.
18. Gabuzda, T. G., Schumann, M. A., Silver, R. K., and Lewis, H. B. (1968). Erythropoietic kinetics in sheep studied by means of induced changes in hemoglobin phenotype. J. Clin. Invest. 47: 1895-1904.
19. DeSimone, J., Biel, M., and Heller, P. (1982). Maintenance of fetal hemoglobin (HbF) elevations in the baboon by prolonged erythropoietic stress. Blood 60: 519-522.
20. Papayannopoulou, T., Torrealba de Ron, A., Veith, R., Knitter, G., and Stamatoyanno-poulos, G. (1984). Arabinosylcytosine induces fetal hemoglobin in baboons by perturbing erythroid cell differentiation kinetics. Science 224: 617-619.
21. Al-Khatti, A., et al. (1987). Stimulation of fetal hemoglobin synthesis by erythropoietin in baboons. N. Engl. J. Med. 317: 415-420.
22. Alter, B., Campbell, A., Holland, J., and Friend, C. (1982). Increased mouse minor hemoglobin during erythroid stress: A model for hemoglobin regulation. Exp. Hematol. 10: 754.
23. Rachmilewitz, E. A., Goldfarb, A., and Dover, G. (1988). Administration of erythropoietin to patients with β-thalassemia intermedia: A preliminary trial. Blood 78: 1145-1147.
24. Olivieri, N. F., Freedman, M. H., Perrine, S. P., Dover, G. J., and Sheridan, B. (1992). Trial of recombinant erythropoietin: Three patients with thalassemia intermedia. Blood 80: 3258-3260.
25. Bourantas, K., Economou, G., and Georgiou, J. (1997). Administration of high doses of recombinant human erythropoietin to patients with beta-thalassemia intermedia-A preliminary trial. Eur. J. Haematol. 58: 22-25.
26. Rachmilevitz, E. A., Aker, M., Perry, D., and Dover, G. (1995). Sustained increase in haemoglobin and RBC following long-term administration of recombinant erythropoietin to patients with homozygous β-thalassemia. Br. J. Haematol. 90: 341-345.
27. Loukopoulos, D., et al. (1998). Hydroxyurea therapy in thalassemia. Ann. N. Y. Acad. Sci. 850: 120-127.
28. Nisli, G., et al. (1997). Recombinant erythropoietin trial in children with transfusion-dependent homozygous beta-thalassemia. Acta Haematol. 98: 199-203.
29. Blau, C. A., et al. (1993). Fetal hemoglobin in acute and chronic states of erythroid expansion. Blood 81: 227-233.
30. Villeval, J. L., et al. (1994). Retrovirus-mediated transfer of the erythropoietin gene in hematopoietic cells improves murine beta thalassemia. Blood 84: 928-933.
31. Dalle, B., et al. (1999). Improvement of mouse β-thalassemia upon erythropoietin delivery by encapsulated myoblasts. Gene Ther. 6: 157-161.
32. Samakoglu, S., et al. (2001). β-Minor globin mRNA accumulation in reticulocytes governs improved erythropoiesis in β-thalassemic mice after erythropoietin cDNA electro-transfer in muscles. Blood 97: 2213-2220.
33. Bohl, D., Bosch, A., Cardona, A., Salvetti, A., and Heard, J. M. (2000). Improvement of erythropoiesis in β-thalassemic mice by continuous erythropoietin delivery from muscles. Blood 95: 2793-2798.
34. Skow, L. C., Burkhart, B. A., and Johnson, F. M. (1983). A mouse model for β thalassemia. Cell 34: 1043-1052.
35. Bohl, D., Salvetti, A., Moullier, P., and Heard, J. M. (1998). Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector. Blood 92: 1512-1517.
36. Alter, B., and Campbell, A. (1984). Increased synthesis of mouse minor hemoglobin in erythroid colonies: A cellular model for hemoglobin regulation. Exp. Hematol. 12: 611-616.
37. Barker, J., Last, J., Adams, S., Nienhuis, A., and Anderson, W. (1973). Hemoglobin switching in sheep and goats: Erythropoietin-dependent synthesis of hemoglobin C in goat bone marrow cultures. Proc. Natl. Acad. Sci. USA 70: 1739-1743.
38. Papayannopoulou, T., Brice, M., and Stamatoyannopoulos, G. (1977). Hemoglobin F synthesis in vitro: Evidence for control at the level of primitive erythroid stem cells. Proc. Natl. Acad. Sci. USA 74: 2923-2927.
39. Alter, B., and Goff, S. (1980). A murine model for the switch from fetal to adult hemoglobin during ontogeny. Blood 56: 1100-1105.
40. Yuan, J., Rubin, E., Aljurf, M., Ma, L., and Schrier, S. (1994). Defective assembly of membrane proteins in erythroid precursors of β-thalassemic mice. Blood 84: 632-637.
41. Mannu, F., et al. (1995). Role of hemichrome binding to erythrocyte membrane in the generation of band-3 alterations in β-thalassemia intermedia erythrocytes. Blood 86: 2014-2020.
42. Yuan, J., Kannan, R., Shinar, E., Rachmilewitz, E., and Low, P. (1992). Isolation, characterization, and immunoprecipitation studies of immune complexes from membranes of β-thalassemic erythrocytes. Blood 79: 3007-3013.
43. Popp, R., Bolch, S., Shinpock, S., and Popp, D. (1990). Expression of the globin genes and hematopoiesis in beta thalassemic mice. Adv. Exp. Med. Biol. 271: 161-165.
44. van den Bos, C., Kieboom, D., Visser, T. P., and Wagemaker, G. (1993). Compensatory splenic hemopoiesis in β-thalassemic mice. Exp. Hematol. 21: 350-353.
45. Sawada, K., et al. (1991). Transitional change of colony stimulating factor requirements for erythroid progenitors. J. Cell. Physiol. 149: 1-8.
46. Gossen, M., and Bujard, H. (1992). Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc. Natl. Acad. Sci. USA 89: 5547-5551.
47. Gossen, M., et al. (1995). Transcriptional activation by tetracyclines in mammalian cells. Science 268: 1766-1769.
48. Dranoff, G., et al. (1993). Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long lasting anti-tumor immunity. Proc. Natl. Acad. Sci. USA 90: 3539-3543.
49. Shinnick, T. M., Lerner, R. A., and Sutcliffe, J. G. (1981). Nucleotide sequence of Moloney murine leukemia virus. Nature 293: 543-548.
50. Salvetti, A., et al. (1998). Factors influencing recombinant adeno-associated virus production. Hum. Gene Ther. 9: 695-706.
51. Grimm, D., and Kleinschmidt, J. A. (1999). Progress in adeno-associated virus type 2 vector production: Promises and prospects for clinical use. Hum. Gene Ther. 10: 2445-2450.
52. Noe, G., Riedel, W., Kubanek, B., and Rich, I. (1999). An ELISA specific for murine erythropoietin. Br. J. Haematol. 104: 838-840.
53. Browne, P., et al. (1997). Removal of erythrocyte membrane iron in vivo ameliorates the pathobiology of murine thalassemia. J. Clin. Invest. 100: 1459-1464.
54. Hoffman-Fezer, G., et al. (1993). Biotin labeling as an alternative nonradioactive approach to determination of red cell survival. Ann. Hematol. 67: 81-87.