Complete Correction of Enzymatic Deficiency and Neurochemistry in the GM1-gangliosidosis Mouse Brain by Neonatal Adeno-associated Virus–mediated Gene Delivery

GM1-gangliosidosis is a glycosphingolipid (GSL) lysosomal storage disease caused by autosomal recessive deficiency of lysosomal acid β-galactosidase (βgal), and characterized by accumulation of GM1-ganglioside and GA1 in the brain. Here we examined the effect of neonatal intracerebroventricular (i.c...

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Veröffentlicht in:	Molecular therapy 2007-01, Vol.15 (1), p.30-37
Hauptverfasser:	Broekman, M L D, Baek, R C, Comer, L A, Fernandez, J L, Seyfried, T N, Sena-Esteves, M
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Animals, Newborn beta-Galactosidase - deficiency beta-Galactosidase - genetics beta-Galactosidase - metabolism Brain Chromatography, High Pressure Liquid Dependovirus - genetics Disease Enzymes Gangliosidosis, GM1 - enzymology Gangliosidosis, GM1 - genetics Gangliosidosis, GM1 - pathology Gangliosidosis, GM1 - therapy Gene therapy Genetic Therapy Lipid Metabolism Lysosomes - enzymology Mice Mice, Inbred C57BL Mice, Knockout Neurochemistry Neurosciences Variance analysis Vectors (Biology)
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