MicroRNA Sets Stage for Human Therapies

The yournal Science reported about a big step toward developing therapies based on naturally occurring tiny RNA molecules called microRNAs. In the first successful experiment with primates, researchers have blocked microRNAs to lower cholesterol levels in monkeys. This achievement builds hope that the strategy could one day be used to attack human diseases.

Scientists have eyed microRNAs as potential targets for disease-fighting treatments because their malfunction has been implicated in cancer, heart disease, viral infections, and other disorders. Ailing mice have recovered thanks to microRNA-based therapies, but researchers have worried whether blocking overactive microRNAs in people will be feasible and safe. The new tests were led by Santaris Pharma, a biotech company in Horsholm, Denmark, focused on developing microRNA-based therapies. The company, which has developed a way to block microRNAs that is different from the strategies previously used in mice, put their method to the test by targeting a microRNA called miR-122 that regulates cholesterol levels.

By blocking tiny pieces of RNA called microRNA in African green monkeys, researchers have shown the potential of using the strategy for fighting disease in humans. Credit: RxGen Inc.

Whole article: Science