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PRIME-RNA: A platform for in vivo preclinical testing of RNA therapeutics

RNA medicines—short strands of nucleic acids that can turn genes “up” or “down”—have changed how we treat brain diseases. For example, nusinersen for spinal muscular atrophy and tofersen for ALS are given by spinal injection to reach the brain and spinal cord. These breakthroughs show that RNA drugs work in the nervous system. However, before these treatments can reach patients, they must be tested in living systems to ensure safety and effectiveness.

Projet en cours Voir le projet et les chercheurs
TYPE DE SUBVENTION
Subventions de la plate-forme
Domaine de recherche
Neurotechnologie
Province
Québec
DATE DE DÉBUT
2026

Neuronal polarity defects as an underlying cause of neurological diseases.

Neurons are cells with long extensions that send information from one part of the nervous system to another.

Projet terminé Voir le projet et les chercheurs
TYPE DE SUBVENTION
Subventions d'équipe
Domaine de recherche
Système nerveux central
Concours
W. Garfield Weston Foundation - Brain Canada Multi-Investigator Research Initiative (MIRI)
Province
Québec
DATE DE DÉBUT
2013