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Titolo:
Advances in experimental treatment of beta-thalassaemia
Autore:
Rodgers, GP; Saunthararajah, Y;
Indirizzi:
NIDDKD, Mol & Clin Hematol Branch, Bethesda, MD 20892 USA NIDDKD BethesdaMD USA 20892 Clin Hematol Branch, Bethesda, MD 20892 USA
Titolo Testata:
EXPERT OPINION ON INVESTIGATIONAL DRUGS
fascicolo: 5, volume: 10, anno: 2001,
pagine: 925 - 934
SICI:
1354-3784(200105)10:5<925:AIETOB>2.0.ZU;2-K
Fonte:
ISI
Lingua:
ENG
Soggetto:
RECOMBINANT-HUMAN-ERYTHROPOIETIN; GLOBIN GENE-EXPRESSION; SICKLE-CELL-ANEMIA; FETAL HEMOGLOBIN INDUCTION; ORAL SODIUM PHENYLBUTYRATE; THALASSEMIA MAJOR; GAMMA-GLOBIN; MARROW TRANSPLANTATION; HISTONE DEACETYLASE; HYDROXYUREA THERAPY;
Keywords:
histone deacetylase inhibitors; methyl-transferase inhibitors; sickle cell disease; thalassaemia; treatment;
Tipo documento:
Review
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
53
Recensione:
Indirizzi per estratti:
Indirizzo: Saunthararajah, Y Univ Illinois, Sect Hematol Oncol MC734, MBRB Rm 3150,900 S Ashland Ave, Chicago, IL 60607 USA Univ Illinois MBRB Rm 3150,900 S Ashland Ave Chicago IL USA 60607
Citazione:
G.P. Rodgers e Y. Saunthararajah, "Advances in experimental treatment of beta-thalassaemia", EXPERT OP I, 10(5), 2001, pp. 925-934

Abstract

beta -Thalassaemia is highly prevalent and world wide in its distribution. The gene to modify the clinical course of patients with transfusion-dependent thalassaemia (thalassaemia major), the gamma -globin gene, is already present in these patients but silenced in the course of development. During erythropoiesis, progenitors are believed to go through a phase where the milieu favours gamma -globin production. One pharmacological strategy to increase gamma -globin production is directed at recruiting such early progenitors through the use of cytotoxic agents (+/- erythropoietin) that presumably deplete more mature progenitors. Another promising strategy is to use chromatin-modifying agents that prevent the silencing of the gamma -globin gene that occurs during development. These agents, the methyl-transferase inhibitors and histone deacetylase inhibitors, either alone or in combination, may be able to produce the robust increase in gamma -globin and hence fetalhaemoglobin and total haemoglobin, needed to successfully treat thalassaemia major. Studies of these agents, which are already available for clinicaltrials, should be encouraged.

ASDD Area Sistemi Dipartimentali e Documentali, Università di Bologna, Catalogo delle riviste ed altri periodici
Documento generato il 20/09/20 alle ore 07:08:06