Our previous research has shown that the ARSACS protein sacsin is essential for proper trafficking of other proteins to the cell surface. In the absence of sacsin this process is disrupted, which may lead to the aberrant localisation of key neuronal proteins, stopping them working properly and contributing to disease. This project aims to use cellular models and analysis of existing human brain gene expression data to better understand which proteins rely on sacsin for normal traffic and function. We will also further investigate the mechanisms by which sacsin loss alters protein trafficking and use advanced computational methods to identify drugs that could potentially correct the trafficking issues. The focus here will be existing drugs that can be repurposed to treat ARSACS.

Financement : 67 900$

Durée : 1ère année d’un projet de 3 ans

Coordonnées:

Dr. Paul Chapple, Faculty of Medicine and Dentistry
Queen Mary University of London

1st Floor North ,John Vane Science Building, Charterhouse Square
London, EC1M 6BQ

 j.p.chapple@qmul.ac.uk