2010 Grants - Robakis

Neuroprotective Functions of Progranulin and Receptor

Nikolaos K. Robakis, Ph.D.
Mount Sinai School of Medicine
New York, New York

2010 Investigator-Initiated Research Grant

Progranulin is a protein that stimulates growth and repair of various cells in the body. Progranulin is also cut into smaller pieces known as granulins, which also regulate cell growth. Recently, a mutation in the gene for progranulin has been shown to account for some cases of frontotemporal lobe degeneration, a neurodegenerative condition affecting the frontal and temporal areas of the brain.

Nikolaos K. Robakis, Ph.D., and colleagues are studying the function of progranulin in the brain. They have preliminary evidence that it activates signaling pathways that protect nerve cells from damage caused by oxidative stress or excessive nerve activity. They plan to confirm and extend these studies to understand how mutations in progranulin can lead to neurodegeneration. Dr. Robakis and colleagues also plan to identify and characterize the receptor for progranulin. As a first step toward this goal, they have already created genetically modified cells that express high levels of progranulin, allowing them to use the protein to bind to and label the receptor. These studies will advance our understanding of the molecular mechanisms involved in frontotemporal degeneration, and may provide insight into possible ways to prevent this disorder.