Genes are segments of DNA (deoxyribonucleic acid) that carry the instructions your body uses to build and operate itself. They are the fundamental units of heredity, passed from parents to offspring.

Cystic fibrosis (CF) gene therapy is a medical approach that aims to treat the underlying genetic cause of cystic fibrosis rather than just managing its symptoms. CF is caused by mutations in the CFTR gene (Cystic Fibrosis Transmembrane Conductance Regulator), which leads to the production of a faulty CFTR protein responsible for regulating salt and water movement in and out of cells. This defect results in thick, sticky mucus buildup in the lungs and other organs.

Gene therapy is a medical technique that involves altering a person’s genes to treat or prevent disease. It aims to correct defective genes responsible for disease development or to introduce new genes into the body to help fight a condition.