The Aga Khan University has received $1.5 million grant from Wellcome Leap to transform treatment of -Thalassemia and Sickle Cell Disease (SCD), which have plagued South Asia, particularly Pakistan. These diseases have not only caused significant suffering but also a heavy economic burden on society. This grant provides a boost to development of innovative gene therapies. Both Thalassemia and SCD are hereditary conditions, arising from genetic anomalies affecting the production of hemoglobin, a vital protein responsible for transporting oxygen in our bloodstream. The research team at AKU aims to explore two advanced gene editing techniques: base editing and prime editing. These techniques have gained popularity due to their precision, specificity and minimal unintended side effects compared to CRISPR-Cas9 technology. “CRISPR is a remarkable technology that allows scientists to make changes in our DNA. Prime editing, however, takes gene editing to next level”, says Dr Afsar Mian, principal researcher of the project. “It offers even more precision and versatility in fixing genetic errors, reduces the chances of unintended effects, and increases effectiveness of the editing process”, he said.