But beyond simply lowering cholesterol, the 82 people with inactive copies also had about half the risk of coronary heart disease as people with two functional copies of the gene. (sciencedaily.com)
In some applications, the AAVs carry functional copies of genes to replace mutated forms present in individuals with genetic diseases. (caltech.edu)
The trial centers on a viral vector, which is used to insert functional copies of the WAS gene into a patient's hematopoietic (blood - forming) stem and progenitor cells, which give rise to all cell types found in the blood and the immune system. (sciencedaily.com)