1. AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann–Pick type C1 disease. (5th June 2018) Authors: Hughes, Michael P; Smith, Dave A; Morris, Lauren; Fletcher, Claire; Colaco, Alexandria; Huebecker, Mylene; Tordo, Julie; Palomar, Nuria; Massaro, Giulia; Henckaerts, Els; Waddington, Simon N; Platt, Frances M; Rahim, Ahad A Journal: Human molecular genetics Issue: Volume 27:Number 17(2018:Sep. 01) Page Start: 3079 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗