AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease
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, FLangue:
english
Journal:
Human Molecular Genetics
DOI:
10.1093/hmg/ddy212
Date:
June, 2018
Fichier:
PDF, 1.86 MB
english, 2018