Manipulation de la réponse neuro-immunitaire par la thérapie génique dans la sclérose en plaques

 

Marie-Ève Janelle

CHU de Québec - Université Laval

 

Domaine : maladies infectieuses et immunitaires

Programme Programme d'appui à la recherche pour les enseignants-chercheurs de collège

Concours 2019-2020

Multiple sclerosis (MS) is an autoimmune disease characterized by the presence of inflammatory lesions and demyelination plaques in the brain and spinal cord that have important consequences for patients, such as disability and suffering. An estimated 75 000 Canadians are living with MS, which is among the highest rates in the world. In patients with MS, the integrity of the blood-brain barrier (BBB) is compromised, allowing immune cells to pass through it and leading to the establishment of an inflammatory environment in the central nervous system (CNS). The cellular and molecular mechanisms underlying CNS inflammation during MS remain unclear. In the search for effective treatments, investigators should use relevant and representative models of the disease. Gene therapy is an approach that uses genes to treat or prevent disease. To date, many clinical trials have shown promising results in various countries, including Canada.

The present research work aims at developing a new gene therapy system that will allow to specifically target the BBB, where inflammation initially takes place and is amplified, in an attempt to reduce brain and spinal cord inflammation and the loss of neurological functions associated with MS. It is expected that this project will allow a better understanding of the MS pathophysiology and could lead to very significant contributions to basic research and even clinical opportunities.