Presentation: P02018

β thalassaemia is caused by mutations in the β globin genes which result in reduced β globin chain synthesis in mature red blood cells. The major pathophysiology, however, is the resulting excess of α globin chains, which precipitate and cause oxidative damage in the cells. This study intends to find agents of therapeutic potential which lower α globin gene expression, therefore reducing the need for blood transfusions in patients with severe forms of the disease. A multiplex cell based assay using an Acumen Explorer fluorescence microplate cytometer was developed to quantitate both α globin expression and cell cytotoxicity during terminal differentiation of red blood cells. Mouse erythroleukaemia stable cell lines which contain the human α globin promoter driving an eGFP reporter were used to measure α globin expression after hexamethylenebisacetamide-induced terminal differentiation. Use of the high content assay for compound screening will be presented.

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