Open access
Jul 2026
Cellular, electrophysiological and behavioral improvements in a mouse model of Rett syndrome following gene therapy combined with focused ultrasound-mediated blood-brain barrier opening.
It is demonstrated that using FUS following systemic administration of an AAV9 vector represents a significant improvement over classical gene therapy protocol for RTT, and the evaluation of the protein levels indicates a possible overdose of Mecp2 protein in the brain cells.
M. Felix, Léna Bourcin, Emilie Borloz et al.
· Experimental Neurology · 0 citations