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

Principal Investigator: ​​​​Dr Afsar ​Mian, Assistant Professor​

​Co-PIs: Dr Hammad Hassan and Dr Salma Jahan

​Funding Source: Grand Challenge Fund of the Higher Education Commission of Pakistan, sponsored by the World Bank

​Duration: July 2021 - June 2024

​​

This pre-clinical research aims to develop druggable gene-editing therapies for beta-thalassemia and sickle cell anaemia. Using CRISPR/Cas9, a molecular gene-editing tool, the researchers 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, the research team at CRM is collaborating with researchers at:

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

Team Members​​

• Dr Fawad Ur Rahman, Postdoctoral Fellow
  

• Dr Muhammad Jameel, Postdoctoral Fellow

• ​Fizza Iftikhar, Postdoctoral Fellow
  

• Dr Irfan Hussain, Postdoctoral Fellow
  

• Syeda Roohina Ali, Postdoctoral Fellow
  

• Zahra Sajid, Research Associate​
  

• Kainaat Mumtaz, Research Associate​​​
  

• Rida e Maria Qazi, Research Associate​​​​​