Gene therapy reduces bad cholesterol by 50% in early trials

Researchers from the University of Barcelona and the University of Oregon have developed an experimental gene therapy strategy that significantly reduces bad cholesterol levels. The method utilizes molecules known as “purine-rich programmable hairpins” (PPRH), which are custom-designed DNA molecules intended to interfere with specific gene activities.

The therapy targets the PCSK9 protein, which plays a key role in regulating low-density lipoprotein cholesterol (LDL-C) levels. By inhibiting this protein, the treatment helps cells clear more cholesterol from the bloodstream, potentially preventing the buildup of fatty plaques in artery walls. In laboratory tests on genetically modified mice, a single injection of the HpE12 molecule reduced plasma protein levels by 50% and total cholesterol levels by 47% by the third day of treatment.

Professor Veronica Noy noted that the molecules reduced protein-specific RNA levels by 74% and protein levels by 87% in tested cells. Compared to existing treatments like statins or monoclonal antibodies, this new technique may offer lower production costs, higher stability, and a lower risk of immune response or muscle-related side effects. However, the study remains in the experimental animal testing phase and requires further clinical trials to evaluate safety and efficacy in humans.