Please use this identifier to cite or link to this item:
http://repository.kln.ac.lk/handle/123456789/23063
Title: | Devising a cure for β-thalassaemia by targeting α-globin |
Authors: | Mettananda, S. |
Keywords: | thalassaemia |
Issue Date: | 2019 |
Publisher: | The Sri Lanka College of Haematologists |
Citation: | The Sri Lanka Journal of Haematology. 2019; 11(1):3-6 |
Abstract: | ABSTRACT: β-Thalassaemia is a disorder of haemoglobin synthesis which does not have an effective cure for a majority of patients affected. Most patients have poor quality of life and die prematurely. The basic pathophysiology of β-thalassaemia is haemolysis and ineffective erythropoiesis due to the imbalance of α-globin chains in red blood cells. Studies done on the molecular pathology and naturally occurring mutations among patients have conclusively shown that decreasing the synthesis of a-globin chains ameliorates the severity of anaemia in β-thalassaemia. A series of recent in vitro and animal studies described in this paper shows that therapeutic inhibition of α-globin synthesis is feasible through genome editing of its major enhancer and pharmacological disruption of epigenetic enzymes. These novel pathways would invariably pave the way for an effective cure for β-thalassaemia which will be available for all patients in the future. |
Description: | Not indexed in MEDLINE |
URI: | http://repository.kln.ac.lk/handle/123456789/23063 |
ISSN: | 1391-7919 |
Appears in Collections: | Journal/Magazine Articles |
Files in This Item:
File | Description | Size | Format | |
---|---|---|---|---|
Devising a cure for beta-thalassaemia.pdf | 1.89 MB | Adobe PDF | View/Open |
Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.