Thalassaemia is a hereditary disease that occurs when a child receives defective genes from both parents. The defect lies in the HBB gene, which is responsible for producing part of the haemoglobin. Without fully functioning haemoglobin, red blood cells break down too quickly, leaving the body oxygen-deprived (TIF, 2021).

Students learn about the daily challenges faced by patients: fatigue, stunted growth, and the constant need for transfusions. Bone marrow transplants can help, but they require a compatible donor, which is rare (Mayo Clinic, 2023).At this point, we move on to the new science of gene therapy. Instead of constantly replacing damaged blood cells, what if we could correct the instructions in the DNA? Scientists are now using tools such as CRISPR-Cas9, often called “genetic scissors”, to cut out the defective genetic sequence and replace it with a healthy version (NIH, 2022). Imagine modifying a recipe that is missing a word so that the dish can finally be prepared correctly. This is exactly what gene therapy does at the molecular level.