Ontology highlight
ABSTRACT:
SUBMITTER: Mettananda S
PROVIDER: S-EPMC6690964 | biostudies-literature | 2019 Aug
REPOSITORIES: biostudies-literature
Mettananda Sachith S Yasara Nirmani N Fisher Christopher A CA Taylor Stephen S Gibbons Richard R Higgs Doug D
Scientific reports 20190812 1
β-Thalassaemia is one of the most common monogenic diseases with no effective cure in the majority of patients. Unbalanced production of α-globin in the presence of defective synthesis of β-globin is the primary mechanism for anaemia in β-thalassaemia. Clinical genetic data accumulated over three decades have clearly demonstrated that direct suppression of α-globin and induction of γ-globin are effective in reducing the globin chain imbalance in erythroid cells hence improving the clinical outco ...[more]