AAV9 intracerebroventricular gene therapy improves...

Human Molecular Genetics 2018 / 06

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, F
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Language:
english
Journal:
Human Molecular Genetics
DOI:
10.1093/hmg/ddy212
Date:
June, 2018
File:
PDF, 1.86 MB
english, 2018
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