familial hypercholesterolemia tagged posts

Gene Therapy using ‘Junk DNA’ could Lower Risk for Heart Disease

Image of LeXis (pink dots) inside a cell. The larger blue dots marks the nuclei of liver cells.

Credit: Judy Wu / UCLA Division of Cardiology Image of LeXis (pink dots) inside a cell. The larger blue dots marks the nuclei of liver cells.

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 a disorder called familial hypercholesterolemia. In a preclinical study, gene LeXis, lowered cholesterol and blockages in the arteries, and the treatment appeared to reduce the build-up of fat in liver cells. Familial hypercholesterolemia is an inherited condition characterized by extremely high levels of LDL and an increased risk of early heart disease.

The LeXis gene belongs to a unique group of genes that until recently were considered “junk DNA” because scientists believed th...

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