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AKU wins grant to develop gene therapies for blood disorders

1 July 2021

Efforts to find a less-invasive and more affordable cure for two common genetic disorders — beta thalassaemia and sickle cell anaemia — have received a boost with the Aga Khan University’s (AKU) research centre recently winning a competitive research grant. “Our team will be among the few researchers around the globe who are working on developing this gene editing approach. Better still, conducting this study in Pakistan will help build local capacity and solutions, instead of waiting for treatments from elsewhere,” said Dr Afsar Mian of AKU’s Centre for Regenerative Medicine and Stem Cell Research (CRM), which will carry out the study. The research will first be tested in the laboratory, using stem cells that have the potential to form any cell type in the body, drawn from thalassaemia and sickle-cell patients. If successful, this will be followed by pre-clinical trials to determine if the treatment is safe.