LAUSR

Intra-cisterna magna delivery of an AAV vector with the GLUT1 promoter in a pig recapitulates the physiological expression of SLC2A1.

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1 Published in 2020 at "Gene therapy"

DOI: 10.1038/s41434-020-00203-z

Abstract: Glucose transporter 1 deficiency syndrome (GLUT1DS) is caused by haplo-insufficiency of SLC2A1, which encodes GLUT1, resulting in impaired hexose transport into the brain. Previously, we generated a tyrosine-mutant AAV9/3 vector in which SLC2A1 was expressed… read more here.

Keywords: aav; intra cisterna; aav glut1; glut1 ... See more keywords