Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 8 of 8 for “"GM2"”.
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Preclinical Assessment of Intravenous Gene Therapy for GM2 Gangliosidosis
GM2 Gangliosidoses are a group of severe neurodegenerative lysosomal storage disorders characterized by the inability to catabolize GM2 Gangliosides, leading to a neurotoxic accumulation of the GM2 lipids within the central nervous system. The Hexosaminidase A (HexA) enzyme is a heterodimeric …
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Phenotypic characterisation of human iPSC neuronal models of GM2 gangliosidoses
… lysosomal storage disorders (LSD). The GM2 gangliosidoses Tay-Sachs and Sandhoff disease are a type of LSD, resulting from the inability of the lysosome to catabolise the breakdown of the ganglioside GM2. This is due to a loss or mutation of either the HEXA or HEXB genes which form the …
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The Role of GM2 Activator Protein in Breast Cancer Metastasis and the EGFR Signaling Pathway
… progression of metastatic disease. The role of GM2 Activator Protein (GM2A), an important transport molecule in ganglioside breakdown, however, has not been thoroughly investigated. In this study, we investigated the role of GM2A protein in metastatic characteristics such as cell proliferation, …
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Correction of AB Variant GM2 Gangliosidoses Using AAV9 Viral Vector Gene Therapy in a Mouse Model
GM2 Gangliosidoses are a group of neurodegenerative diseases affecting the brain. In humans, these diseases are characterized by rapid neurological deterioration and death before 4-years of age. GM2 gangliosides are normally degraded in a cell’s lysosomes through the action of three gene products, …
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Partial Molecular and Biochemical Characterization of a Sandhoff Mouse Model
… neurodegeneration. SD is caused by a build-up of GM2 Gangliosides, a lipid which is stored in the lysosome of neurons. It can occur in infantile, juvenile and adult forms; in the severe, infantile form, death occurs by the age of 4. It is well understood that this lipid build-up is the result of a …
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Analyzing A-series gangliosides in neurons following exposure to glutamate
… while no significant changes were observed for GM2 and GM3 expression. Furthermore, neurons that were pretreated with GM1 showed increased viability compared to untreated neurons when exposed to glutamate. Immunofluorescence revealed an elevated expression of GM3 in activated microglia compared …
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Identification and characterization of an enterocyte receptor for group A porcine rotavirus
… chromatographic mobility between that of GM2 and GM3. Inhibition of blocking activity by treatment of GMX with neuraminidase and ceramide glycanase, but not by treatment with protease or heat (100$\sp\circ$C) supported the identification of GMX as a ganglioside. Further purification of GMX …
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The role of heterozygous lysosomal storage disorder alleles as risk factors for dementia
… respectively. Nor did it lead to increased GM2 ganglioside (the substrate degraded by the enzyme encoded by Hexb), or related gangliosides, GM1 or GM3. Surprisingly, heterozygosity of Hexb resulted in less amyloid beta plaques in the orbital cortex and hippocampus of 46-week-old …