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Pharmacological Manipulation of Fetal Hemoglobin Synthesis in Patients with Severe β‐Thalassemia

1985; Wiley; Volume: 445; Issue: 1 Linguagem: Inglês

10.1111/j.1749-6632.1985.tb17189.x

1749-6632

Arthur W. Nienhuis, Timothy J. Ley, R. Keith Humphries, Neal S. Young, George J. Dover,

Neonatal Health and Biochemistry

Annals of the New York Academy of SciencesVolume 445, Issue 1 p. 198-211 Pharmacological Manipulation of Fetal Hemoglobin Synthesis in Patients with Severe β-Thalassemia ARTHUR W. NIENHUIS, Corresponding Author ARTHUR W. NIENHUIS Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219a Address correspondence to: Arthur W. Nienhuis, M.D., Building 10, Room 7C103, National Institutes of Health, Bethesda, MD 20205.Search for more papers by this authorTIMOTHY J. LEY, TIMOTHY J. LEY Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219Search for more papers by this authorR. KEITH HUMPHRIES, R. KEITH HUMPHRIES Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219Search for more papers by this authorNEAL S. YOUNG, NEAL S. YOUNG Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219Search for more papers by this authorGEORGE DOVER, GEORGE DOVER Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219Search for more papers by this author ARTHUR W. NIENHUIS, Corresponding Author ARTHUR W. NIENHUIS Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219a Address correspondence to: Arthur W. Nienhuis, M.D., Building 10, Room 7C103, National Institutes of Health, Bethesda, MD 20205.Search for more papers by this authorTIMOTHY J. LEY, TIMOTHY J. LEY Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219Search for more papers by this authorR. KEITH HUMPHRIES, R. KEITH HUMPHRIES Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219Search for more papers by this authorNEAL S. YOUNG, NEAL S. YOUNG Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219Search for more papers by this authorGEORGE DOVER, GEORGE DOVER Clinical Hematology Branch National Heart, Lung, and Blood Institute Bethesda, Maryland 20205 Department of Pediatrics Johns Hopkins University Baltimore, Maryland 21219Search for more papers by this author First published: June 1985 https://doi.org/10.1111/j.1749-6632.1985.tb17189.xCitations: 28AboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookTwitterLinkedInRedditWechat REFERENCES b1 Nienhuis, A. W. & L. Wolfe. 1985. The thalassemias: Disorders of hemoglobin synthesis. In Hematology of Infancy and Childhood. W. B. Saunders. Philadelphia , PA . In press b2 Nienhuis, A. W., N. P. Anagnou & T. Ley. 1984. Advances in thalassemia research. Blood 63: 738–758 b3 Humphries, R. K., T. Ley, P. Turner, A. D. Moulton & A. W. Nienhuis. 1982. Differences in α-, β, and δ-globin gene expression in monkey kidney cells. Cell 30: 173–183 b4 Van der Ploeg, L. H. T. & R. A. Flavell. 1980. DNA methylation in human γδ-β-locus in erythroid and nonerythroid tissues. Cell 19: 947–958 b5 DeSimone, J., P. Heller, L. Hall & D. Zwiers. 1982. 5-Azacytidine stimulates fetal hemoglobin (Hb F) synthesis in anemic baboons. Proc. Natl. Acad. Sci. USA 79: 428–4431 b6 Ley, T. J., J. DeSimone, N. P. Anagnou, G. H. Keller, R. K. Humphries, P. H. Turner, N. S. Young, P. Heller & A. W. Nienhuis. 1982. 5-Azacytidine selectively increases γ-globin synthesis in a patient with β+-Thalassemia. N. Engl. J. Med. 307: 1469–1475 b7 Ley, T. J., J. DeSimone, C. T. Noguchi, P. H. Turner, A. N. Schechter, P. Heller & A. W. Nienhuis. 1983. 5-Azacytidine increases γ-globin synthesis and reduces the proportion of dense cells in patients with sickle cell anemia. Blood 62: 370–380 b8 Charache, S., G. Dover, K. Smith, C. C. Talbot, M. Moyer & S. Boyer. 1983. Treatment of sickle cell anemia with 5-azacytidine results in increased fetal hemoglobin production and is associated with nonrandom hypomethylation of DNA around the β-globin gene complex. Proc. Natl. Ad. Sci. USA 80: 4842–4846 b9 Torrealba-de Ron, A. T., Th. Papayannopoulou, M. S. Knapp, M. Feng-Ruen Fu, G. Knitter & G. Stamatoyannopoulos. 1984. Perturbations in erythroid marrow progenitor cell pools may play a role in the augmentation of Hb F by 5-azacytidine. Blood 63: 201–210 b10 Papayannopoulou, Th., A. T. Torrealba-de Ron, R. Veith, R. Knitter & G. Stamatoyannopoulos. 1984. Arabinosylcytosine induces fetal hemoglobin in baboons by perturbing erythroid cell differentiation kinetics. Science 224: 617–619 b11 Letvin, N. L., D. G. Linch, G. P. Beardsley, K. W. McIntyre & D. G. Nathan. 1984. Augmentation of fetal-hemoglobin production in anemic monkeys by hydroxyurea. N. Engl. J. Med. 310: 869–874 b12 Platt, O. S., S. H. Orkin, G. Dover, G. P. Beardsley, B. Miller & D. G. Nathan. 1984. Hydroxyurea enhances fetal hemoglobin production in sickle cell anemia. J. Clin. Invest. 74: 652–656 b13 Dover, G., S. H. Boyer & W. R. Bell. 1978. Microscopic methods for assaying F-cell production: Illustrative changes during infancy and aplastic anemia. Blood 52: 664–672 b14 Humphries, R. K., G. Dover, N. S. Young, J. Moore, S. Charache, T. Ley & A. W. Nienhuis. 1984. 5-Azacytidine acts directly on both exythroid precursors and progenitors to increase Hb F production. J. Clin. Invest. In press b15 Treisman, R., N. J. Proudfoot, M. Shander & T. Maniatis. 1982. A single-base change at a splice site in a β°-thalassemia gene causes abnormal RNA splicing. Cell 29: 903–911 b16 Goldsmith, M. E., R. K. Humphries, T. J. Ley, A. Kline, J. Kantor & A. W. Nienhuis. 1983. Silent nucleotide substitution in a β+-thalassemia globin gene activates splice site in coding sequence RNA. Proc. Natl. Acad. Sci. USA 80: 2318 b17 Ley, T. J., N. P. Anagnou, G. Pepe & A. W. Nienhuis. 1982. RNA Processing Errors in Patients with β-thalassemia. Proc. Natl. Acad. Sci. USA 79: 4775 b18 Sands, J., A. Macher, T. Ley & A. W. Nienhuis. 1984. Disseminated infection caused by Cunninghamella bertholletiae in a β-thalassemia patient receiving 5-azacytidine. Ann. Intern. Med. In press b19 Finch, C. A., K. Deubelbeiss, J. D. Cook, J. W. Bchbach, L. A. Harker, D. D. Funk, G. Marsaglia, R. S. Hillman, S. Slichter, J. W. Adamson, I. A. Ganzon & E. R. Giblett. 1970. Ferrokinetics in man. Medicine 49: 17–53 b20 Dover, G., S. Charache & G. Vogelsang. 1983. 5-azacytidine increases hemoglobin F production in sickle cell anemia: Results of 300 days of therapy. Blood 62 (Suppl. 1): 55a b21 Byrd, A. T. 1984. DNA methylation—how important in gene control? Nature 307: 503–504 b22 Dover, G. J. & G. H. Wyer. 1980. Quantitation of hemoglobins within individual red cells: Asynchronous biosynthesis of fetal and adult hemoglobin during erythroid maturation in normal subjects. Blood 56: 1082–1091 b23 Wood, W. G. & R. W. Jones. 1981. Erythropoiesis and hemoglobin production: A unifying model involving sequential gene activation. In hemoglobins and Development in Differentiation. G. Stamatoyannopoulos & A. W. Nienhuis, Eds. Alan R. Liss, Inc. New York b24 Papayannopoulou, Th., T. Kalmantis & G. Stamatoyannopoulos. 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 b25 Ley, T. J., Y. L. Chiang, D. Haidaris, N. P. Anagnou, V. L. Wilson & W. F. Anderson. 1984. DNA methylation and regulation of the human β-like globin genes in mouse erythroleukemia cells in human chromosome 11. Proc. Natl. Acad. Sci. USA 81: 6618–6622 Citing Literature Volume445, Issue1Fifth Cooleyís Anemia SymposiumJune 1985Pages 198-211 ReferencesRelatedInformation