DNA therapy cuts PCSK9 and lowers cholesterol in mice, hinting at a statin-free option
Researchers used polypurine hairpins (PPRHs) to suppress PCSK9 gene expression, increasing LDL receptors. In human liver cells HpE12 reduced PCSK9 RNA by 74% and protein by 87%. In transgenic mice, a single HpE12 dose lowered plasma PCSK9 by 50% and cholesterol by 47% after three days, suggesting a potential statin-free approach that still needs safety testing in humans.