Mécanismes de dysfonctionnement synaptique dans un modèle génétique de l'amyotrophie spinale et développement de thérapies

 

Kessen (Shunmoogum) Patten

Institut national de la recherche scientifique [INRS]

 

Domaine :  neurosciences, santé mentale et toxicomanies

Programme chercheurs-boursiers - Junior 1

Concours 2017-2018

Spinal muscular atrophy (SMA) is an incurable disease of the motoneurons caused by defects in the gene, survival of motor neuron (SMN). We aim to understand how loss of SMN leads to defective connectivity at peripheral and central synapses and to identify small molecules that can rescue this connectivity and motor function in a fish model of SMA. We will examine the strength of connectivity at all levels within the motor circuit using cutting-edge genetic and physiological techniques. In addition, we will screen chemical compound libraries for lead compounds that can reverse/ameliorate motor defects in our SMN-depleted fish model.This proposed research plan will enhance our understanding of synaptic pathology in SMA and may accelerate the development of therapeutics for this devastating disease.