The present invention relates to a transformed stem cell expressing the GLB1 gene and a pharmaceutical composition for treating G M1 gangliosidosis or type IVB mucopolysaccharide including the same, the present invention relates to a nasal cavity of a stem cell transformed with a GLB1 gene When injected into, the stem cells are delivered to the cranial nerve tissue and can express β-galactosidase-1 enzyme. Specifically, the present invention was to optimize the type of gene introduction method and lentiviral expression vector to produce a stem cell with improved expression of the GLB1 gene. In addition, the treatment efficiency was improved by optimizing the number of cells administered intranasally. Under the optimized conditions, the transformed stem cells expressing the GLB1 gene into the nasal cavity of the G M1 gangliosidosis-induced mice were confirmed to significantly reduce the G M1 gangliosides accumulated in the cranial nerve tissue. Therefore, the transformed stem cells expressing the GLB1 gene of the present invention, and the pharmaceutical composition for treating G M1 gangliosidosis or type IVB mucopolysaccharide, comprising the same well in β-galactosidase-1 enzyme in brain tissue It can be effectively used to treat G M1 gangliosidosis or type IVB mucopolysaccharide, since it can effectively degrade G M1 gangliosides that have been expressed and accumulated in cerebral nerve tissue.
展开▼