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Studying the involvement of cell surface RNA binding proteins in ALS pathogenesis

Project ongoing

Project Overview

Amyotrophic lateral sclerosis (known as ALS or Lou Gehrig’s disease) is a debilitating disease that causes loss of control over muscles and leads to death quickly after diagnosis. In Canada, each year approximately 1000 people die of ALS and a similar number of people are newly diagnosed with this disease. It remains incurable, as the molecular defects causing this disease are still poorly understood. What many ALS cases have in common are malfunctions in the systems which cells use to properly transport and use factors crucial for the transfer of genetic information: RNAs and their sidekicks, RNA-binding proteins. Recent work showed that these factors can be localized in an unexpected place, the surface of human cells. There, these factors could be helping the cells recognize other cells, communicate with them, and push them to make certain decisions. This project aims to shed light on the roles of these cell surface localized RNA factors and explore their impact to affect disease progression in the case of ALS. Improving our understanding of the RNA world localized at the cell surface could help inform future therapeutic strategies to interrupt undesirable cell-to-cell communication that is the hallmark of this terrible disease.

Partners and Donors

ALS Canada