AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann–Pick type C1 disease
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E. Henckaerts | F. Platt | A. Rahim | S. Waddington | David A Smith | A. Colaco | Giulia Massaro | Mylene Huebecker | Lauren Morris | Michael P. Hughes | C. Fletcher | J. Tordo | N. Palomar | G. Massaro | M. Hughes | Alexandria Colaco