Gene-editing trials show 52% reduction in LDL cholesterol

Researchers in China and the United States have reported promising results from gene-editing trials aimed at treating high cholesterol. In a study conducted by Shanghai Jiao Tong University, an experimental single-dose injection targeting the PCSK9 gene reduced LDL cholesterol levels by 52% in volunteers with familial hypercholesterolemia over a six-month period. The treatment utilizes a modified enzyme derived from the Hafnia paralvei bacterium to weaken the gene's ability to produce cholesterol.

In a separate study by the Cleveland Clinic in the United States, researchers targeted the ANGPTL3 gene. Participants receiving the highest dose saw a 52% reduction in LDL cholesterol and a 48% reduction in triglycerides.

These gene-editing technologies aim to provide a long-term solution for patients who currently rely on daily statin medications to manage cholesterol levels. While some participants reported mild side effects such as fever and muscle aches, no dangerous adverse effects were observed. Clinical protocols include long-term monitoring of participants for up to 15 years.