For the first time, researchers have produced evidence that gene-editing can cut high cholesterol, a major risk factor for the nation’s leading killer.
Preliminary results from a study involving 10 patients born with a genetic condition that causes very high cholesterol found that editing a gene inside the liver can significantly reduce levels of “bad cholesterol.”
The experimental treatment needs to be tested on more patients who would be followed for much longer to confirm the approach is safe and effective. But the results are being hailed as a potential landmark proof-of-concept that could eventually provide a powerful new way to prevent heart attacks and strokes.
Researchers used a form of gene-editing known as CRISPR, which enables scientists to make very precise changes in DNA much more easily than ever before. Specifically, they used a newer version of CRISPR known as “base-editing,” which gives scientists the power to rewrite individual letters in the genetic code.
In this case, the editing occurred in liver cells in the organ inside the body. Other approaches to gene-editing have required removing cells from the body, editing them in the lab and then infusing them back into patients.
