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Showing 1 to 1 of 1 for “"AB-variant GM2 gangliosidoses"”.
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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, …