“Scalable therapeutic testing in the ARSACS mouse” – Drs. Justin Wolter & Huaxia Wang

Our project aims to accelerate drug discovery for ARSACS by using advanced single nucleus RNA sequencing (snRNAseq) to test 8 therapeutic candidates in the ARSACS mouse model (including ongoing studies led by Drs. Gentil/Durham, and Drs. Lim/Schmahmann). Unlike traditional methods that rely on variable and lengthy behavioral tests, our approach directly measures molecular and cellular changes in key brain cell types affected by ARSACS, providing more precise and scalable assessments of drug effects. By identifying how each drug alters gene expression and cell populations linked to disease progression, we can prioritize the most promising treatments for further development. This work aims to streamline and improve preclinical testing, ultimately helping to bring effective therapies to the ARSACS community faster.

Grant: $100,000

Duration: first year of a 3 year project

This project has been jointly funded with the Richardson Trust Fund who made a 50 000$ contribution.