Key Findings and Potential
The study, involving 15 volunteers, demonstrated that one dose of the drug could safely reduce both total cholesterol and levels of harmful triglycerides by about half. These exciting results were presented at the American Heart Association's annual meeting and published in The New England Journal of Medicine.
"Rather than a lifetime worth of medicine, we have the potential to give people a cure," said Dr. Luke Laffin, a preventative cardiologist at the Cleveland Clinic who helped conduct the research.
If confirmed in larger trials, this revolutionary approach could be a powerful new tool in the fight against heart disease, the leading cause of death in the US. It could free millions from the daily routine of taking statins and other cholesterol-lowering drugs, addressing the "huge" problem of patients quitting their medications, noted Dr. Steven Nissen, another Cleveland Clinic cardiologist involved in the study.
How the Treatment Works
The drug is infused into the bloodstream and travels to the liver to disable a gene called ANGPTL3, which is responsible for producing cholesterol and triglycerides. "It's a knockout of the gene. It cuts it. And after that, the gene no longer functions," Dr. Nissen explained.
Samarth Kulkarni, CEO of CRISPR Therapeutics, the drug's developer and study sponsor, believes this approach "could potentially impact millions of people around the world."
Cautions and Future Steps
While the findings are encouraging and consistent with similar research, experts emphasize that much more research is needed to confirm the drug's long-term safety and effectiveness.
Cardiologists caution that currently, the idea of a simple, "one-and-done" treatment remains a "fantasy" because gene editing is expensive, and its long-term safety is still unclear. The high cost of other gene therapies—sometimes reaching millions per patient—is a significant barrier.
Furthermore, scientists like Dr. Kiran Musunuru of the University of Pennsylvania note that the safety bar will be much higher for a treatment given to otherwise healthy people to prevent future illness.
Researchers are now planning larger, longer studies to determine if this one-time gene-editing drug can safely protect people against heart attacks and strokes for a lifetime.
Disclaimer: This content is published only for health awareness and informational purposes. It's not a substitute for your professional medical advice. You must consult a doctor/healthcare professional regarding your specific health concerns.
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