LDL receptor antisense oligonucleotide

LDL receptor antisense oligonucleotide (LDLR ASO) is a synthetic oligonucleotide designed to knock down the expression of the low-density lipoprotein receptor (LDLR). By binding to LDLR mRNA, the ASO promotes its degradation, thereby reducing the number of LDL receptors on the cell surface. While reducing LDLR is typically associated with the pathology of familial hypercholesterolemia in humans, this ASO is primarily utilized as a research tool in preclinical studies to create inducible and reversible mouse models of hypercholesterolemia and atherosclerosis. This approach allows researchers to study both the progression and regression of atherosclerotic plaques by controlling the timing of LDLR knockdown and subsequent recovery upon cessation of treatment.

Other names

LDLR ASOLow-density lipoprotein receptor antisense oligonucleotide