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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 Irfan Hussain, Dr Ambrin Fatima, Dr Salman Kirmani​

​Funding Source: Wellcome Leap 

​Duration: August 2023 - June 2025

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 Mureed Hussain, Postdoctoral Fellow

  • Dr Shanzay Ahmed, Postdoctoral Fellow

  • ​Dr Hafiz Muhammad Ghufran, Postdoctoral Fellow

  • Dr Rida -e- Maria Qazi, Postdoctoral Fellow​

  • Shagufta Naz​​, Research Associate​

  • Maliha Javed, Research Associate​​​

  • Hijab Zahra, Research Associate​​​​​

  • Anwar Alam, Research Associate​​​​​

  • Safana Farooq, PhD Student ​


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