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

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

​Co-PIs: Dr Fawad ur Rehman​,  Dr Ambrin Fatima, Dr Salman Kirmani​

​Funding Source: Wellcome Leap 

​Duration: August 2023 - February 2026

In recent years, base editing and prime editing have become prominent tools for gene editing due to their precise targeting, minimal unintended effects, and high success rates.

This project aims to harness these cutting-edge techniqu​es to correct mutations in the beta-globin (HBB) gene responsible for β-thalassemia and sickle cell disease. To do this, our researchers will isolate and grow haematopoietic stem cells in the laboratory. The genetic error will be corrected or fixed first in the laboratory, thereby restoring the production of healthy hemoglobin. Once successful, the therapy will be tested on animal models before introduction to clinical trials. ​

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​​Team Members​​

  • Dr Rida -e- Maria Qazi, Senior Instructor

  • Maliha Javed, Research Associate​​​

  • Dr Ayaz Khan, Post-doctoral Fellow

  • Anwar Alam, Research Associate​​​​​

  • Safana Farooq, PhD Student ​


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