Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis

Adeno-associated virus 2-mediated gene therapy decreases autofluorescent storage material and increases brain mass in a murine model of infantile neuronal ceroid lipofuscinosis

Infantile neuronal ceroid lipofuscinosis (INCL) is the earliest onset form of a class of inherited neurodegenerative disease called Batten disease. INCL is caused by a deficiency in the lysosomal enzyme palmitoyl protein thioesterase-1 (PPT1). Autofluorescent storage material accumulates in virtuall...

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Bibliographic Details
Main Authors: Megan Griffey, Ellen Bible, Carole Vogler, Beth Levy, Praveena Gupta, Jonathan Cooper, Mark S Sands
Format: Article
Language:English
Published: Elsevier 2004-07-01
Series:Neurobiology of Disease
Subjects:
Infantile neuronal ceroid lipofuscinosis
Lysosomal storage disease
Batten disease
Gene therapy
Neurodegenerative disease
Adeno-associated virus
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996104000634

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http://www.sciencedirect.com/science/article/pii/S0969996104000634

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