Prof Nicole Déglon
Head of Neurosciences Research Center (CRN)
Head of the Laboratory of Laboratory of Cellular and Molecular Neurotherapies
Laboratoire des neurothérapies cellulaires et moléculaires - LNCM
Keywords: Neurodegenerative diseases, Huntington’s disease, Gene therapy, Gene editing
The group has a long-standing experience and expertise in viral gene transfer technology to deliver therapeutic candidates in the brain or to model CNS pathologies by overexpressing disease-causing proteins.
We focus our research on the development of molecular therapies for neurodegenerative disorders and in particular huntingtin (HTT) gene editing for Huntington’s disease (HD). We have been exploiting the unique features and targeting specificities of viral vectors to deliver therapeutic candidates, generate new models of CNS pathologies or improve our understanding of the pathological mechanisms. In parallel, we are taking advantage of local and cell-type specific overexpression of transgenes in the CNS to investigate spreading of wild-type Tau protein in sporadic tauopathies as well as the contribution of mitochondrial dysfunctions in early AD.