Gene Editing Shows Promise in Lowering Cholesterol, potential to Prevent Heart Disease
A new study indicates that CRISPR gene-editing technology can effectively reduce levels of cholesterol and triglycerides, offering a potential one-time treatment for cardiovascular disease.researchers are cautiously optimistic about the findings, though meaningful hurdles remain before the therapy becomes widely available.
The treatment, developed by CRISPR Therapeutics, involves infusing a drug into the bloodstream that travels to the liver and disables the ANGPTL3 gene, which plays a role in cholesterol and triglyceride production. “It’s a knockout of the gene. it cuts it.And after that, the gene no longer functions,” explained Dr. steven Nissen, a preventive cardiologist at the Cleveland Clinic involved in the research.
Early results are encouraging, mirroring similar work being done by Verve Therapeutics in Boston.”The fact that we now have more clinical data that ther’s a ’there there’ is of course tremendously encouraging,” said Fyodor Urnov, a gene editing researcher at the University of California, Berkeley. “Having a CRISPR medicine for heart attack would be an extraordinary win.”
Though, experts emphasize the need for further research. dr. Eric Topol, a cardiologist at Scripps Research, noted that gene-editing is “expensive, long-term safety is unclear.” Dr. Kiran Musunuru, scientific director of the Center for Inherited Cardiovascular Medicine at the University of Pennsylvania perelman School of Medicine, agreed, stating, “It could be a very important tool, but to actually prove it’s protective against cardiovascular disease you need to do more study.” He also pointed out that safety standards would be higher for use in otherwise healthy individuals.
Currently, millions rely on daily medication to manage cholesterol, but heart disease remains the leading cause of death in the U.S., claiming nearly 700,000 lives annually. A significant challenge is patient adherence to long-term medication regimens, as highlighted by Dr. Nissen: “This problem of adherence – that people stop taking their medications – is huge.”
CRISPR Therapeutics CEO Samarth Kulkarni believes the approach “could possibly impact millions of people around the world.” While the cost of the treatment remains undisclosed,other gene-editing therapies have proven extremely expensive,potentially costing millions per patient. Researchers are planning larger, longer-term studies to assess the long-term safety and efficacy of the gene-editing treatment in preventing heart attacks and strokes.
