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

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 under the control of the endogenous GLUT...

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Veröffentlicht in:	Gene therapy 2021-06, Vol.28 (6), p.329-338
Hauptverfasser:	Nakamura, Sachie, Osaka, Hitoshi, Muramatsu, Shin-ichi, Takino, Naomi, Ito, Mika, Jimbo, Eriko F., Watanabe, Chika, Hishikawa, Shuji, Nakajima, Takeshi, Yamagata, Takanori
Format:	Artikel
Sprache:	eng
Schlagworte:	42 42/44 631/208 631/208/199 Animals Antibodies Ataxia Biomedical and Life Sciences Biomedicine Care and treatment Catheters Cell Biology Cerebrospinal fluid Cisterna Magna Cytomegalovirus Dependovirus - genetics Diagnostic tests Endothelium Gene Expression Gene Therapy Genetic vectors Genetic Vectors - genetics Genomes Glucose Glucose transporter Glucose Transporter Type 1 - genetics Health aspects Hexose Hogs Human Genetics Injection Intellectual disabilities Metabolism, Inborn errors of Methods Mice Mutants Nanotechnology Neuronal-glial interactions Physiology Plasmids Promoters (Genetics) Proteins Swine Transgenes Tyrosine Vectors (Biology) Viruses
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