Leveraging proximity interactomics to identify therapeutically targetable RNA-binding proteins in G4C2 repeat-driven ALS
Amyotrophic lateral sclerosis (ALS) is a progressive disease that causes loss of control over muscles and leads to death quickly after diagnosis. It remains incurable, with a subtype of ALS being commonly associated with a specific genetic mutation that causes a toxic RNA molecule to be produced. This repeat-containing RNA is thought to clog the cell’s transport systems and impact cell functioning. This project aims to understand how the repeat RNA is inducing the transport difficulties, which could help develop strategies to “unclog” the cells and restore their normal information flow, potentially laying the groundwork for the development of therapeutic approaches to treat this terrible disease.
Ana Novacic , Institut de recherches cliniques de Montréal (ICRM)
Partners and Donors
Linda Auger Morissette and Friends