Improvement of erythropoiesis in β-thalassemic mice by continuous erythropoietin delivery from muscle

Blood

Volumn 95, Issue 9, 2000, Pages 2793-2798

  Bohl, Delphine ^b   Bosch, Assumpció ^b   Cardona, Ana ^b   Salvetti, Anna ^b   Heard, Jean Michel ^a  

^a INSTITUT PASTEUR   (France)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

ERYTHROPOIETIN; GLOBIN; HEMOGLOBIN F; IRON 59;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BETA THALASSEMIA; CONTINUOUS INFUSION; CONTROLLED STUDY; DRUG EFFICACY; GLOBIN SYNTHESIS; HORMONAL THERAPY; IRON KINETICS; MOUSE; NONHUMAN; PRIORITY JOURNAL; RETICULOCYTE COUNT;

EID: 0034194044     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v95.9.2793.009k07_2793_2798     Document Type: Article

References (39)

1. Scott M, Van den Berg J, Repka T, et al. Eflect of excess alpha-hemoglobin chains on cellular and membrane oxidation in model β-thalassemic erythrocytes. J Clin Invest. 1993;91:1706-1712.
2. Schrier S. Pathobiology of thalassemic erythrocytes. Current Op Hematol. 1997;4:75-78.
3. Muretto P, Angelucci E. Images in clinical medicine. Successful treatment of severe thalassemia. N Engl J Med. 1999;341:92.
4. Boulad F, Giardina P, Gillio A, et al. Bone marrow transplantation for homozygous β-thalassemia. Ann N Y Acad Sci. 1998;850:498-502.
5. Weinberg RS, Schofield JM, Lenes AL, Brochstein J, Alter BP. Adult "fetal-like" erythropoiesis characterizes recovery from bone marrow transplantation. Br J Haematol. 1986;63:415-424.
6. Papayannopoulou T, Nute PE, Kurachi S, Stamatoyannopoulos G. Consistent activation of fetal hemoglobin synthesis in cultured adult bone marrow cells. Blood. 1978;51:671-679.
7. Chui DH, Wong SC, Enkin MW, Patterson M, Ives RA. Proportion of fetal hemoglobin synthesis decreases during erythroid cell maturation. Proc Natl Acad Sci U S A. 1980;77:2757-2761.
8. Umemura T, Al-Khatti A, Papayannopoulou T, Stamatoyannopoulos G. Fetal hemoglobin synthesis in vivo: direct evidence for control at the level of erythroid progenitors. Proc Natl Acad Sci USA. 1988;85:9278-9282.
9. Rachmilewitz EA, Goldfarb A, Dover G, Administration of erythropoietin to patients with beta-thalassemia intermedia: a preliminary trial. Blood. 1988;78:1145-1147.
10. Olivieri NF, Freedman MH, Perrine SP, Dover GJ, Sheridan B. Trial of recombinant erythropoietin: three patients with thalassemia intermedia. Blood. 1992;80:3258-3260.
11. Bourantas K, Economou G, Georgiou J. Administration of high doses of recombinant human erythropoietin to patients with beta-thalassemia intermedia: a preliminary trial. Eur J Haematol. 1997;58:22-25.
12. Rachmilevitz EA, Aker M, Perry D, Dover G. Sustained increase in haemoglobin and RBC following long-term administration of recombinant erythropoietin to patients with homozygous β-thalassemia. Br J Haematol. 1995;90:341-345.
13. Nisli G, Kavakli K, Aydinok Y, Oztop S, Cetingul N, Basak N. Recombinant erythropoietin trial in children with transfusion-dependent homozygous beta-thalassemia. Acta Haematol. 1997;98:199-203.
14. Loukopoulos D, Voskaridou E, Stamoulakatou A, et al. Hydroxyurea therapy in thalassemia. Ann N Y Acad Sci. 1998;850:120-127.
15. Kessler PD, Podsakoff GM, Chen X, et al. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA. 1996;93: 14,082-14,087.
16. Bohl D, Naffakh N, Heard JM. Long term control of erythropoietin secretion levels by tetracycline in mice transplanted with engineered primary myoblasts. Nature Med. 1997;3:299-312.
17. Bohl D, Salvetti A, Moullier P, Heard JM. Control of erythropoietin delivery by doxycycline in mice after intramuscular injection of adeno-associated vector. Blood. 1998;92:1512-1517.
18. Rendhal KG, Left SE, Otten GR, et al. Regulation of gene expression in vivo following transduction by two separate rAAV vectors. Nat Biotechnol. 1998;16:757-761.
19. Ye X, Rivera M, Zoltick P, et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science. 1999;283:88-91.
20. Skow LC, Burkhart BA, Johnson FM. A mouse model for β thalassemia. Cell. 1983;34:1043-1052.
21. Advani R, Rubin E, Mohandas N, Schrier S. Oxidative red blood cell membrane injury in the pathophysiology of severe mouse β-thalassemia. Blood. 1992;79:1064-1067.
22. Whitney JB. Differential control of the synthesis of two hemoglobin beta chains in normal mice. Cell. 1977;12:863-871.
23. Alter BP, Goff SC. A murine model for the switch from fetal to adult hemoglobin during ontogeny. Blood. 1980;56:1100-1105.
24. Alter BP, Campbell AS. Increased synthesis of mouse minor hemoglobin in erythroid colonies: a cellular model for hemoglobin regulation. Exp Hematol. 1984;12:611-616.
25. Alter B, Campbell A, Holland J, Friend C. Increased mouse minor hemoglobin during erythroid stress: a model for hemoglobin regulation. Exp Hematol. 1982;10:754-760.
26. Alter BP, Wagner CK. A murine model for hemoglobin regulation: hydroxyurea increases mouse minor hemoglobin in vivo. Prog Clin Biol Res. 1987;251:479-485.
27. Leroy-Viard K, Rouyer-Fessard P, Beuzard Y. Improvement of mouse β-thalassemia by recombinant human erythropoietin. Blood. 1991;78:1596-1602.
28. Samulski RJ, Chang LS, Shenk T. A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication. J Virol. 1987;61: 3096-3101.
29. Salvetti A, Orève S, Chadeuf G, et al. Factors influencing recombinant adeno-associated virus production. Human Gene Trier. 1998;9:695-706.
30. Dodge JT, Mitchell C, Hanahan DJ. The preparation and chemical characteristics of hemoglobin-free ghosts of human erythrocytes. Arch Biochem Biophys. 1963;100:119-130.
31. Yuan J, Rubin E, Aljurf M, Ma L, Schrier S. Defective assembly of membrane proteins in etythroid precursors of β-thalassemic mice. Blood. 1994; 84:632-637.
32. Snyder RO, Spratt SK, Lagarde C, et al. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Human Gene Ther. 1997;8: 1891-1900.
33. Duan D, Sharma P, Yang J, et al. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue. J Virol. 1998;72:8568-8577.
34. Papayannopoulou T, Brice M, Stamatoyannopoulos G. Hemoglobin F synthesis in vitro: evidence for control at the level of primitive erythroid stem cells. Proc Natl Acad Sci U S A. 1977;74:2923-2927.
35. Papayannopoulou T, Kalmantis T, Satmatoyannopoulos G. 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. 1979;76:6420-6424.
36. Udupa KB, Reissmann KR. In vivo erythropoietin requirements of regenerating erythroid progenitors (BFU-e, CFU-e) in bone marrow of mice. Blood. 1979;53:1164-1171.
37. Popp R, Bolch S, Shinpock S, Popp D. Expression of the globin genes and hematopoiesis in beta thalassemic mice. Adv Exp Med Biol. 1990; 271:161-165.
38. Weinberg RS, Antonarakis SE, Kazazian HH, et al. Fetal hemoglobin synthesis in erythroid cultures in hereditary persistence of fetal hemoglobin and beta 0-thalassemia. Blood. 1984;63: 1278-1284.
39. Dalle B, Payen E, Regulier E, et al. Improvement of mouse β-thalassemia upon erythropoietin delivery by encapsulated myoblasts. Gene Therapy. 1999;6:157-161.