Understanding α-globin gene regulation and implications for the treatment of β-thalassemia.

dc.contributor.authorMettananda, S.
dc.contributor.authorGibbons, R.J.
dc.contributor.authorHiggs, D.R.
dc.date.accessioned2016-01-20T09:38:56Z
dc.date.available2016-01-20T09:38:56Z
dc.date.issued2016
dc.description.abstractOver the past three decades, a vast amount of new information has been uncovered describing how the globin genes are regulated. This knowledge has provided significant insights into the general understanding of the regulation of human genes. It is now known that molecular defects within and around the α- and β-globin genes, as well as in the distant regulatory elements, can cause thalassemia. Unbalanced production of globin chains owing to defective synthesis of one, and the continued unopposed synthesis of another, is the central causative factor in the cellular pathology and pathophysiology of thalassemia. A large body of clinical, genetic, and experimental evidence suggests that altering globin chain imbalance by reducing the production of α-globin synthesis ameliorates the disease severity in patients with β-thalassemia. With the development of new genetic-based therapeutic tools that have a potential to decrease the expression of a selected gene in a tissue-specific manner, the possibility of decreasing expression of the α-globin gene to improve the clinical severity of β-thalassemia could become a reality.en_US
dc.identifier.citationAnnals of the New York Academy of Sciences.2016;1368(1):16-24en_US
dc.identifier.issn0077-8923 (Print)
dc.identifier.issn1749-6632 (Electronic)
dc.identifier.urihttp://repository.kln.ac.lk/handle/123456789/11255
dc.language.isoen_USen_US
dc.publisherWiley-Blackwellen_US
dc.subjecttreatment of ß-thalassemiaen_US
dc.titleUnderstanding α-globin gene regulation and implications for the treatment of β-thalassemia.en_US
dc.typeArticleen_US

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