This project aims to discover new therapeutic targets and potential drugs for ARSACS by combining blood samples from patients with advanced iPSC (induced pluripotent stem cell) technology and artificial intelligence (AI). The team will analyze gene expression patterns in blood samples, patient-derived iPSCs, and genetically corrected control cells to identify common disease mechanisms across different SACS mutations. Using Oncocross’s RAPTOR AI platform, approximately 25,000 drug candidates will be screened to find compounds that could reverse ARSACS-associated cellular changes.
Grant: $25,000
Duration : one year
Dr. Nicolas Dupré
LOEX, Aile-R, R-111
Hôpital de l’Enfant-Jésus
1401, 18e Rue
Québec (Québec) G1J 1Z4
Contact: nicolas.dupre.med@ssss.gouv.qc.ca