Silencing silencers to boost foetal globin production and treat β-haemoglobinopathies [ 2019 - ]

Research Grant

Brief description 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.

Funding Amount $560,394.00

Funding Scheme Project Grants

Notes Standard Project Grant