Silencing silencers to boost foetal globin production and treat β-haemoglobinopathies

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The genetic disorders β-thalassaemia and sickle cell anaemia are caused by mutations in the adult globin gene. Fortunately, humans have a second globin gene, the foetal γ-globin gene, which is expressed in utero but turned off after birth. Re-activating expression of this foetal globin gene can alleviate the symptoms of β-thalassaemia and sickle cell anaemia. We are using cutting edge gene editing technology to explore strategies for boosting foetal globin expression in affected individuals.