Autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) is a rare inherited disease that damages the nervous system and muscles, caused by faults in a protein called sacsin. There is currently no cure, and existing treatments only ease some symptoms. To search for better options, we took skin cells from ARSACS patients, reprogrammed them into brain cells carrying the same faulty protein, and tested many drugs to see if they could reverse the damage. After promising early results, we are now studying exactly how these drugs work, how safe they are, and whether they can reverse symptoms in laboratory models. We are also investigating how well these drugs can reach the brain — a key hurdle in treating neurological diseases.
Financement: 100 000$
Ce projet a été financé conjointement avec le Richardson Trust Fund qui a contribué 50 000$ à ce projet.
Durée : un an
Dr Mohan Babu
3737 Wascana Parkway
University of Regina, Regina
Saskatchewan S4S OA2
Coordonnées: mohan.babu@uregina.ca