The goal of this proposal is to validate the misregulation of Tau in ARSACS and establish a platform from which to explore therapeutic targeting of proteins involved in tau pathology as a treatment for ARSACS. Grant : $98,758 Contact: Dr. Anthony Hickey, Director of...
Targeting deregulated calcium homeostasis as potential therapeutic option for ARSACS -Dr. Francesca Maltecca
The major aims of this project are: 1) to evaluate the functional role of sacsin at the synaptic level, in particular its involvement in the control of calcium fluxes in Purkinje neurons at the postsynaptic space; 2) to strengthen the evidence of efficacy of an...
Is SARM1 a therapeutic target for ARSACS?- Dr. Thomas Schwarz
This project uses the sacsin mutant mouse and mice lacking SARM1 to ask whether the progression of the cell loss in ARSACS can be slowed or prevented by removing SARM1. A positive outcome would make ARSACS a strong candidate for this therapeutic strategy. Duration :...
Is ARSACS a Tauopathy?- Dr. Paul Chapple
The objectives of the project will be published at a later date Duration: 3 year funded project Grant: 32,827 pounds Contact: Dr.Paul Chapple, Professor of Molecular Cell BiologyCentre for Endocrinology Barts and The London, Queen Mary’s School of Medicine and...
Towards the Structural Determination of Sacsin- Dr. Walid A. Houry
Aim 1. Engineering cells with tagged SACS for purification Aim 2. Purification and determination of SACS structure Aim3: To purify and determine the structure of Canadian SACS mutants Duration : 2 year project Grant: $74,000 in year 1Contact: Dr. Walid A. Houry,...
Elucidating Mechanisms Underlying Motor Coordination Rescue in a Mouse Model of ARSACS – Dr. Alanna Watt
Aim 1 Identifying druggable Targets for potential ARSACS treatment Aim 2 Characterize pathophysiology for future druggable target development Duration : 2nd year of the funded projectGrant: $75,000Contacts: Dr. Alanna Watt,Department of Biology McGill...
Elucidating Mechanisms Underlying Motor Coordination Rescue in a Mouse Model of ARSACS – Dr. Anne McKinney
Aim 1 Identifying druggable Targets for potential ARSACS treatment Aim 2 Characterize pathophysiology for future druggable target development Duration : 2nd year of the funded projectGrant: $50,000Contact: Dr. Anne McKinney, Department of Pharmacology and...
Sacin’s role in Synapses: Early Pathological Changes in ARSACS- Dr. Bernard Brais
Aim 1: Sacsin’s role in dendritic spines morphology Aim 1.1. Assess the impact of sacsin loss on dendritic spines morphology Aim 1.2. Assess the role of sacsin partners on dendritic spine morphology. Aim 1.3. Study spine morphology in Sacs-/- mice....
Optimization of Active Molecules Towards a Pre-Clinical in Vivo Proof of Mechanism of ARSACS Phenotype Reversal – Dr. Michel Bouvier
The goal is to develop small molecules that reverse the cellular phenotype of ARSACS into a drug that would stop the progression of the disease and/or lead to clinical improvement. Grant: $160,000 Contact Dr.Michel Bouvier, Institute for Research in Immunology and...
Therapeutic Approaches for ARSACS- Dr. Benoit Gentil
To obtain preclinical proof-of-concept for ARSACS treatments based on: 1) a protein/gene replacement approach and 2) drug treatment using HDAC inhibitors. Grant: $100,000 Contact Dr. Benoit Gentil, Department of Kinesiology and Physical Education, McGill...