Novel ASO therapeutic target designed against hyperlipidemia via PCSK9 knockdown

With the gradual improvement of individuals' living standards, there has been a concurrent escalation in the consumption of fats and sugars in the daily dietary habits. Consequently, an increasing number of individuals are afflicted by hyperlipidemia, a condition that, could elevate blood viscosity, thereby engendering serious complications in a long run. Traditional lipid-lowering medications, such as statins, manifest substantial side effects, thereby imposing a significant metabolic burden on the liver and kidneys. Conversely, antisense oligonucleotides (ASOs) exhibit attributes such as rapid absorption, prolonged efficacy, and minimal side effects. In light of these considerations, a novel ASO was meticulously designed, sebsequently, its efficacy and toxicity assessments were conducted both in vitro and in vivo. The results unequivocally demonstrate the effectiveness and safety of this ASO. [Display omitted] •A potential gene therapy site for hyperlipidemia has been discovered.•ASOs have been synthesized, validated, and tested for toxicity to knock down PCSK9.•ASO52-L96 may contribute to treat seborrheic alopecia.