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Background
The delivery of adeno‐associated virus (AAV) vectors via the cerebrospinal fluid (CSF) has emerged as a valuable method for widespread transduction in the central nervous system. Although infusion into the cerebral ventricles is a common protocol in preclinical studies of small animals, the cisterna magna has been recognized as an alternative target for clinical studies because it can...
Background
We generated an adeno‐associated virus (AAV) vector in which the human SLC2A1 gene, encoding glucose transporter type 1 (GLUT1), was expressed under the human endogenous GLUT1 promoter (AAV‐GLUT1). We examined whether AAV‐GLUT1 administration could lead to functional improvement in GLUT1‐deficient mice.
Methods
We extrapolated human endogenous GLUT1 promoter sequences from rat minimal...
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