Scientists from UCLA and the Howard Hughes Medical Institute successfully used a gene that suppresses cholesterol levels as part of a treatment to reduce plaque in mice with familial hypercholesterolemia. They found that the gene, LeXis, lowered cholesterol and blockages in the arteries, and the treatment appeared to reduce the build-up of fat in liver cells. Their results were reported last week in the journal Circulation.
The team tested whether a single injection of LeXis could slow the development of heart disease. They gave the mice either LeXis or a control gene, and fed them a 15-week diet consisting of food high in sodium and cholesterol—the mouse equivalent of fast-food hamburgers and french fries. Researchers then measured the progression of heart disease.
Although previous research has shown that lncRNAs like LeXis can be important, according to the team, this is the first study to show that they could potentially be used to treat a human disease using gene therapy. In the next phase of the study, researchers intend to confirm the findings in larger animals and test the therapy in combination with currently available treatments.
Caption: LeXis (pink dots) inside a cell. The larger blue dots marks the nuclei of liver cells. Image courtesy of Judy Wu / UCLA Division of Cardiology