Team “Gene Therapy” - Institut du Cerveau

Scientific Teams

Team “Gene Therapy”

Presentation

Gene therapy Main domain: Clinical & translational neurosciences Subdomain : Molecular & cellular neuroscience Nathalie CARTIER’s team develops gene therapy strategies for severe neurodegenerative diseases including Huntington's disease, Spinocerebellar ataxias, Alzheimer's disease and genetic leukodystrophies. Research includes proof of concept in animal models and translational steps to clinical applications. The team aims to propose a phase I/II therapeutic trial in 2020 for Huntington disease. The team will also develop tools for the delivery of therapeutic molecules in the brain (use of microglia, optimization of AAV for intravenous administration, optogenerapy for time-controlled delivery).

Major publications

Djelti F, J Braudeau, E Hudry, M Dhenain, J Varin, I Bièche, C Marquer, F Chali, S Ayciriex, N Auzeil, S Alves, D Langui, MC Potier, O Laprevote, M Vidaud, C Duyckaerts, R Miles, P Aubourg*, N Cartier* CYP46A1 inhibition, brain cholesterol accumulation and neurodegeneration pave the way for Alzheimer’s disease. Brain 2015 : 138 : 2383-98
Burlot MA, Braudeau J, Michaelsen-Preusse K, Potier B, Ayciriex S, Varin J, Gautier B, Djelti F, Audrain M, Dauphinot L, Fernandez-Gomez FJ, , Laprévote O, Bièche I, Auzeil N, Potier MC, Dutar P, Korte M, Buée L, Blum D, Cartier N Cholesterol24-hydroxylase defect is implicated in memory impairments of Alzheimer-like Tau pathology Hum Mol Genet. 2015 : 24 : 5965-76.
Fol R, Braudeau J, Ludewig S, Abel T, Weyer SW, Roederer JP, Brod F, Audrain M, Bemelmans AP, Buchholz CJ, Korte M, Cartier N*#, Müller UC*#. Viral gene transfer of APPsα rescues synaptic failure in an Alzheimer’s disease mouse model. 2015 Nov 4
Boussicault L*, * Alves S, Lamazière A, Planques A, Heck N, Moumné L, Despres G, Bolte S, -Hue A, Pagès C, Galvan L, Piguet F, Aubourg P, Cartier N*, Caboche J*and Betuing S* . CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington’s disease. Brain 2016 Mar;139(Pt 3):953-70
Alves S, Churlaud G, Audrain M, Michaelsen-Preusse K, Fol R, Braudeau J, Korte M, Klatzmann D*§, Cartier N*§ Interleukin-2 improves amyloid pathology, synaptic failure and memory in Alzheimer’s disease mice. 2017 Mar 1;140(3):826-842. doi: 10.1093/brain/aww330.