Gene therapy for a mouse model of glucose transporter-1 deficiency syndrome

Objective: We generated an adeno-associated virus (AAV) vector in which the human SLC2A1 gene was expressed under the synapsin I promoter (AAV-hSLC2A1) and examined if AAV-hSLC2A1 administration can lead to functional improvement in GLUT1-deficient mice. Methods: AAV-hSLC2A1 was injected into hetero...

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Main Authors: Sachie Nakamura, Hitoshi Osaka, Shin-ichi Muramatsu, Naomi Takino, Mika Ito, Shiho Aoki, Eriko F. Jimbo, Kuniko Shimazaki, Tatsushi Onaka, Sumio Ohtsuki, Tetsuya Terasaki, Takanori Yamagata
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Molecular Genetics and Metabolism Reports
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Online Access:http://www.sciencedirect.com/science/article/pii/S2214426916301318