​Gene Editing Therapies for Beta-Thalassemia and Sickle Cell Anaemia​

Team Lead: ​​​​Dr Afsar ​Mian​​, Assistant Professor​

​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 all​ow the defective part of the HBB gene to be sniped and repaired, enabling the production of normal haemoglobin.​

​The second therapy involves reactivating the production of foetal haemoglobin as a substitute for the deficient adult haemoglobin in beta-thalassemia and sickle cell anaemia. Dr Mian and his team will work on gene-silencing, using the same gene-editing tool and approach, suppressing the BCL11A gene that stops foetal haemoglobin from being produced.​

For this project, Dr Mian and his team are collaborating with researchers at:

  • University of California, San Francisco
  • Norwegian University of Science and Technology​
  • Cardiff University in the UK​​​​

Team Members​