AKU Receives $1.5 M Boost to Revolutionize Treatment of β-Thalassemia and SCD
Summary
Aga Khan University has received a $1.5 million grant from Wellcome Leap, that will support a project that seeks to transform the treatment of -Thalassemia and Sickle Cell Disease (SCD), which have plagued South Asia, particularly Pakistan. Sadly, the current treatment options are challenging to sustain, with less than one percent of patients surviving beyond the age of 20.Thalassemia varies worldwide, with over 300 different mutations in various regions, including Pakistan. Due to this diversity, the increasing focus on personalized treatments or therapies developed in one country may not effectively address the unique challenges faced by patients in another. Thus, localized research is crucial to understand region-specific mutations and develop tailored solutions.Dr Afsar Mian, Assistant Professor at Aga Khan University, and the principal investigator of the project, explains the limitations of the only known curative therapy, allogenic hematopoietic stem cell (HSC) transplantation, which is dependent on donor compatibility, is expensive and can result in treatment failure. 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.