MicroRNAs' Role In Neurodegeneration

For the first time, researchers have shown that microRNAs—the short, noncoding pieces of RNA that fine-tune protein production in cells—are key players in a neurodegenerative disease (Nature 2010, 466, 637). Stanford University School of Medicine’s Bingwei Lu and colleagues report that the leucine-rich repeat kinase 2 (LRRK2) mutation, which increases the risk of developing Parkinson’s disease, alters LRRK2’s interaction with two specific microRNAs. This disruption results in overproduction of E2F1 and DP, two proteins that are toxic to the dopamine-producing neurons that die off in Parkinson’s. Lu’s team showed that reducing the level of these two proteins prevents development of Parkinson’s symptoms in fruit flies, which are often used as model organisms in biochemical research. “Many pharmaceutical companies are already making compounds that act on these two proteins, which in previous studies have been shown to be associated with cancer,” Lu says. “It may be possible to take these compounds off the shelf or quickly adapt them for use in noncancer indications such as Parkinson’s.”