US, China trials suggest a single nanodrug shot might stop bad cholesterol forever
Separate trials using CRISPR and base editing to silence lipid genes show drastic drops in LDL-C without daily medication Imagine swapping your daily cholesterol pill for a single injection that keeps “bad” cholesterol under control for a year, a decade – or even a lifetime. Two clinical trials, one in the United States and another in China, suggest that this one-and-done approach could become a reality. Low-density lipoprotein cholesterol (LDL-C), better known as “bad” cholesterol, is a major driver of heart disease – the leading cause of death worldwide. Triglycerides, another type of blood fat, add to the risk. For millions of people, the standard fix is a daily statin pill. Research led by the Cleveland Clinic in the US and carried out in Australia, New Zealand and Britain, tested a gene-editing nanodrug called CTX310 on 15 volunteers. The therapy uses tiny particles to carry a CRISPR gene-editing tool straight into liver cells. Its target is a gene named ANGPTL3, which helps control how much fat circulates in the blood. Some people are born with a natural mutation that inhibits ANGPTL3. They have extremely low cholesterol and triglycerides, and they rarely develop heart disease.
