The team has succeeded in creating the first representative animal model of 4H leukodystrophy, one of the common forms of the disease. This is a crucial step in the development of treatments, and was recently described in an article published in the journal"This significant progress will allow us to understand the disease better and to test putative therapies, which we have already started doing.
"It was essential to create a representative model of the disease to develop therapeutic approaches in the laboratory in future preclinical studies," explains Dr. Bernard, who is also associate professor in the Department of Neurology and Neurosurgery, and Pediatrics, at McGill University."The lab is now working on the development of the first gene replacement therapy approach for the disease.
"This discovery is a critical building block in the quest to discover effective treatments for people affected by 4H and demonstrates the advances that are possible when patients, families, and researchers come together to identify the most important problems to solve and find the right people to solve them.
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