Gene therapy using ‘junk DNA’ could lower risk for heart disease
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, researchers 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. Familial hypercholesterolemia is an inherited condition characterized by extremely high levels of low-density lipoprotein cholesterol (commonly referred to as "bad cholesterol") 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 they served little purpose. However, evidence from the human genome project led to the discovery that genes like LeXis are actually active. The study of these genes, now referred to as long noncoding ribonucleic a...