A shot against heart attacks?
Harvard Stem Cell Institute (HSCI) scientists collaborating with researchers at the University of Pennsylvania have developed a “genome-editing” approach for permanently reducing cholesterol levels in mice through a single injection, a development that could reduce the risk of heart attacks in humans by 40 to 90 percent.
“For the first iteration of an experiment, this was pretty remarkable,” said Kiran Musunuru of HSCI, an assistant professor in Harvard’s Department of Stem Cell and Regenerative Biology (SCRB), and a cardiologist at Harvard-affiliated Brigham and Women’s Hospital. Musunuru stressed, however, that it could take a decade of concerted effort to get this new approach for fighting heart disease from the laboratory to phase I clinical trials in humans.
The research was published online today by Circulation Research, a journal of the American Heart Association. Qiurong Ding, a postdoctoral fellow in Musunuru’s laboratory, is first author on the paper. The work by the Musunuru team and a Penn team led by Daniel J. Rader, an authority on cholesterol metabolism and a long-time collaborator of Musunuru, focused on altering the function of a liver gene called PCSK9.
In 2003, a group of researchers in France studying families with very high cholesterol levels and very early heart attacks discovered that PCSK9 was a cholesterol regulator, because they found that mutations in this gene seemed to be responsible for the high cholesterol levels and the heart attacks. These mutations are extremely rare and are limited to a few families.