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Lrp2 ASO is an antisense oligonucleotide designed to reduce the expression of the LRP2 gene, which encodes megalin, a multiligand endocytic receptor mainly expressed in the proximal tubule of the kidney. Lrp2 ASO works by targeting LRP2 mRNA, leading to its degradation and suppressed protein synthesis. This pharmacological approach aims to modulate megalin abundance and thereby affect processes such as protein reabsorption and potentially atherosclerosis development. Lrp2 ASO has been investigated in animal models, notably for its effects on hypercholesterolemia-induced atherosclerosis, by systemically reducing LRP2/megalin protein and subsequently suppressing atherosclerosis without inducing severe renal dysfunction[5]. The drug's primary research focus is preclinical and early translational studies targeting cardiovascular and renal disease mechanisms.
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