Over 35 million Americans take daily medications to reduce their cholesterol, and that number continues to increase. But thanks to new research from the Harvard Stem Cell Institute and the University of Pennsylvania, it’s possible that patients will be able to experience an improved quality of life with a single injection!
By disrupting gene activity in a gene (PCSK9) that regulates cholesterol, researchers were able to permanently reduce cholesterol by 35-40%. First, they targeted the DNA sequence where the gene resides, then created a break in the system, and then used adenovirus to carry the treatment to the liver. In one injection, they were able to permanently change the genome, meaning that the benefits are there forever.
While this treatment is probably at least 5-10 years away for humans, the accomplishment in mice is pretty amazing. The next step in this research is to work with mice that have human-derived liver cells before moving into human studies. Read more about it here: