Gene therapy for Glut1 -deficient mouse using an adeno-associated virus vector with the human intrinsic GLUT1 promoter

Brain microvasculature defects and Glut1 deficiency syndrome averted by early repletion of the glucose transporter-1 protein

(2017) Maoxue Tang et al.   Nature Communications

Outcome of ketogenic diets in GLUT1 deficiency syndrome in Japan: A nationwide survey

(2016) Tatsuya Fujii et al.   BRAIN & DEVELOPMENT

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

(2016) Joana Saraiva et al.   JOURNAL OF CONTROLLED RELEASE

Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

(2016) Haiyan Fu et al.   Molecular Therapy-Methods & Clinical Development

AAV9-mediated central nervous system–targeted gene delivery via cisterna magna route in mice

(2016) Vera Lukashchuk et al.   Molecular Therapy-Methods & Clinical Development

Tailored transgene expression to specific cell types in the central nervous system after peripheral injection with AAV9

(2016) Jonathan Dashkoff et al.   Molecular Therapy-Methods & Clinical Development

Nationwide survey of glucose transporter-1 deficiency syndrome (GLUT-1DS) in Japan

(2015) Yasushi Ito et al.   BRAIN & DEVELOPMENT

Triheptanoin dramatically reduces paroxysmal motor disorder in patients with GLUT1 deficiency

(2015) Fanny Mochel et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Glucose metabolism transporters and epilepsy: Only GLUT1 has an established role

(2014) Michael S. Hildebrand et al.   EPILEPSIA

GLUT1 deficiency syndrome into adulthood: a follow-up study

(2014) W. G. Leen et al.   JOURNAL OF NEUROLOGY

Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2delivery to motor neurons

(2013) Takenari Yamashita et al.   EMBO Molecular Medicine

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

(2013) S J Gray et al.   GENE THERAPY

GLUT1 deficiency syndrome 2013: Current state of the art

(2013) Valentina De Giorgis et al.   SEIZURE-EUROPEAN JOURNAL OF EPILEPSY

Systemic Delivery of Tyrosine-Mutant AAV Vectors Results in Robust Transduction of Neurons in Adult Mice

(2013) Asako Iida et al.   Biomed Research International

Cerebrospinal Fluid Analysis in the Workup of GLUT1 Deficiency Syndrome

(2013) Wilhelmina G. Leen et al.   JAMA Neurology

Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency

(2012) W.-L. Hwu et al.   Science Translational Medicine

A modified Atkins diet is promising as a treatment for glucose transporter type 1 deficiency syndrome

(2011) YASUSHI ITO et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Fibroblast Growth Factor 21 Induces Glucose Transporter-1 Expression through Activation of the Serum Response Factor/Ets-Like Protein-1 in Adipocytes

(2011) Xuan Ge et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Glucose transporter deficiency syndrome (GLUT1DS) and the ketogenic diet

(2008) Jörg Klepper   EPILEPSIA

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY