LeXis gene therapy (University of California Los Angeles/Hughes Medical Institute)

This article describes about long noncoding RNA facilitated gene therapy reduces atherosclerosis in murine model of familial hypercholesterolemia.AAV8 vector-expressing LeXis under control of human liver-specific thyroxine-binding globulin promoter.Treatment with AAV8.hTBG.LeXis led to sustained expression of LeXis and significant reduction of Srebp2 and its target genes involved in cholesterol biosynthesis, including Hmgcr in mouse liver.Examination of serum lipids at the end of the study showed significant decrease in total cholesterol and triglyceride levels in LeXis-treated mouse.Feasibility of lncRNA mimetic therapeutic strategy for intervention in a chronic metabolic disease such as atherosclerosis.The author concluded that gene therapy-facilitated expression of lncRNA was feasible, and chronic LeXis production in liver reduces serum cholesterol and atherosclerosis in mouse model of familial hypercholesterolemia.