Gene Therapy Halves Bad Cholesterol in Trial

A groundbreaking gene-editing therapy developed by researchers in China has demonstrated significant success in reducing high cholesterol levels. In a small trial involving six volunteers, a single dose of the experimental jab led to a substantial decrease in "bad" cholesterol within six months.

This therapy is being developed for familial hypercholesterolemia, a severe inherited disease that elevates blood fat levels and drastically increases heart attack risk. The treatment targets the PCSK9 gene in the liver, which is crucial for removing LDL-C. By weakening this gene's activity, the therapy aims to improve the liver's cholesterol-clearing capability.

Patients who received the highest dose experienced a 74% reduction in PCSK9 enzyme activity and a 52% drop in "bad" cholesterol. Mild side effects, such as fever and muscle aches, were reported but resolved quickly, with no other adverse events noted. Long-term follow-up for up to 15 years is planned.

In parallel research, a US trial targeting the ANGPTL3 gene also showed promising results, with significant reductions in both bad cholesterol and triglycerides. Scientists are hopeful that these gene-editing advancements could eventually be expanded to treat a wider range of lipid disorders.