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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Detalhes bibliográficos
Publicado no:Mol Genet Metab Rep
Main Authors: Nakamura, Sachie, Osaka, Hitoshi, Muramatsu, Shin-ichi, Takino, Naomi, Ito, Mika, Aoki, Shiho, Jimbo, Eriko F., Shimazaki, Kuniko, Onaka, Tatsushi, Ohtsuki, Sumio, Terasaki, Tetsuya, Yamagata, Takanori
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2017
Assuntos:
Research Paper
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5238605/
https://ncbi.nlm.nih.gov/pubmed/28119822
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2016.12.008
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