Treatment agent for lysosomal storage disease
AssigneesFUJIFILM Corporation, Kyoto Prefectural Public University Corporation
InventorsKentaro Nakamura, Tsukasa Kitahashi, Rie Hando, Satoshi Gojo, Daisuke Kami
Life of this patent
Estimating the term…
Abstract
An object of the present invention is to provide an excellent treatment agent for a lysosomal storage disease. According to the present invention, there is provided a treatment agent for a lysosomal storage disease including a cell structure which includes a plurality of biocompatible polymer blocks and a plurality of cells of at least one type and in which at least one of the polymer blocks is disposed in gaps between the plurality of cells.
Claims19
A method of treating a lysosomal storage disease, comprising transplanting a cell structure to a subject in need of a treatment for a lysosomal storage disease, wherein the cell structure includes a plurality of biocompatible polymer blocks and a plurality of mesenchymal stem cells, further wherein at least one of the polymer blocks is disposed in gaps between the plurality of mesenchymal stem cells,
- wherein the cells do not overexpress a gene necessary for a therapy of a lysosomal storage disease by introduction of the gene,
- wherein the number of mesenchymal stem cells transplanted by the cell structure is 1.0×105 cells/kg to 2.0×108 cells/kg, and
- wherein the number of cells to be administered to the subject is 1.0×105 cells/kg to 2.0×108 cells/kg,
- provided that the lysosomal storage disease is not metachromatic leukodystrophy, Hurler syndrome, or Hunter syndrome.
A method of treating Fabry disease, comprising transplanting a cell structure to a subject in need of a treatment for Fabry disease, wherein the cell structure includes a plurality of biocompatible polymer blocks and a plurality of fibroblast cells, wherein at least one of the polymer blocks is disposed in gaps between the plurality of fibroblast cells,
- wherein the cells do not overexpress a gene necessary for a therapy of Fabry disease by introduction of the gene,
- wherein the number of mesenchymal stem cells and/or fibroblasts transplanted by the cell structure is 1.0×105 cells/kg to 2.0×108 cells/kg, and
- wherein the number of cells to be administered to the subject is 1.0×105 cells/kg to 2.0×108 cells/kg.
Dependencies are inferred from the claim text.