Gene Editing Therapies for Beta-Thalassemia and Sickle Cell Anaemia
Funding Source: Grand Challenge Fund of the Higher Education Commission of Pakistan, sponsored by the World Bank
Duration: July 2021 - June 2024
Through this pre-clinical research, Dr Afsar Mian and his team aim to develop druggable gene-editing therapies for beta-thalassemia and sickle cell anaemia. Using CRISPR/Cas9, a molecular gene-editing tool, Dr Mian and his team aspire to fix a mutation in the haemoglobin beta-globin (HBB) gene that causes both these disorders. This new therapy could be injected as a drug to allow the defective part of the HBB gene to be sniped and repaired, enabling the production of normal haemoglobin.