GLB1 GM1 TRANSFORMED STEM CELL EXPRESSING GLB1 GENE AND PHARMACEUTICAL COMPOSITION FOR TREATING GM1 GANGLIOSIDOSIS COMPRISING SAME

摘要

The present invention relates to a transformed stem cell capable of expressing GLB1 gene, and a pharmaceutical composition for G_M1 gangliosidosis or IVB-type mucopolysaccharidosis (MPS) treatment including the transformed stem cell. When a stem cell with transformed GLB1 gene is injected into the nasal cavity, the stem cell is transmitted to the nerve tissue in the brain and can express β-galaktosidase-1 enzyme. To be more specifically, the stem cell manufactured by the present invention can have an improved expression ratio of GLB1 gene by optimizing a gene introducing method and the type of a lentivirus expression vector. In addition, the stem cell can improve treatment efficiency by optimizing the number of cells inputted into the nasal cavity. Thus, it is verified that G_M1 ganglioside accumulated inside the nerve tissue in the brain is significantly reduced when the transformed stem cell capable of expressing GLB1 gene is injected into the nasal cavity of a G_M1 gangliosidosis causing mouse under the optimized condition. Therefore, the transformed stem cell capable of expressing GLB1 gene, and the pharmaceutical composition for G_M1 gangliosidosis or IVB-type MPS treatment including the transformed stem cell can be useful in treatment of G_M1 gangliosidosis or IVB-type MPS by well expressing β-galaktosidase-1 enzyme in the nerve tissue in the brain and effectively disintegrating G_M1 ganglioside accumulated in the nerve tissue in the brain.