“Dimethyl Fumarate Tested on the ARSACS Mouse Model” – Drs. Wolter & Wang
This project, led by Drs. Justin Wolter and Huaxia Wang, aims to test the therapeutic potential of dimethyl fumarate (DMF) in the ARSACS mouse model.
Tuesday, November 21, 2023

This project, led by Drs. Justin Wolter and Huaxia Wang, aims to test the therapeutic potential of dimethyl fumarate (DMF) in the ARSACS mouse model. DMF is currently approved for the treatment of multiple sclerosis (MS), where it has been shown to reduce neuroinflammation, oxidative stress, and neuronal death. Additionally, DMF has been tested in preclinical models of other neurodegenerative diseases, such as amyotrophic lateral sclerosis, Parkinson’s disease, and Alzheimer’s disease, where it demonstrates promising neuroprotective effects. In our study, we will evaluate the therapeutic potential of DMF in the ARSACS mouse model, testing motor function and molecular markers of neuroinflammation, oxidative stress, and neurodegeneration. Successful results could pave the way for DMF-based treatments in future ARSACS clinical trials, and determine the utility of specific biomarkers in tracking ARSACS progression.
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New Funding
“Modelling ARSACS in human in iPSC-derived cerebellar organoids ” – Dr. Esther Becker
This project aims to create three-dimensional disease models called ‘organoids’ using induced pluripotent stem cells (iPSCs) from ARSACS patients.

New Funding
“Structural Determination of Sacsin” – Dr. Walid Houry
We have carried out early in vitro characterizations of Sacsin, and we are working to obtain its three-dimensional (3D) structure using cryogenic electron microscopy.