Composition for use in treating dystrophic epidermolysis bullosa
AssigneeOsaka University
InventorsKatsuto TAMAI, Yasushi KIKUCHI, Tomoki TAMAKOSHI
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Abstract
The present disclosure relates to a composition for use in the treatment of dystrophic epidermolysis bullosa, comprising a cell obtained from a patient with dystrophic epidermolysis bullosa, wherein the cell is a mesenchymal stem cell and genetically modified to produce type VII collagen. The present disclosure also relates to a composition for use in the treatment of dystrophic epidermolysis bullosa, comprising a cell that produces type VII collagen, wherein the composition is to be administered into a blister.
Claims21
1-16. (canceled)
A method of treating dystrophic epidermolysis bullosa, comprising the steps of:
- (a) obtaining mesenchymal stem cells from a patient with dystrophic epidermolysis bullosa;
- (b) genetically modifying the cells to produce type VII collagen;
- and
- (c) administering the cells of (b) to the patient.
A method of treating dystrophic epidermolysis bullosa, comprising:
- administering a therapeutically-effective amount of cells which produce type VII collagen to a patient in need thereof, the administering being intrablister administration.
A gRNA comprising a sequence selected from the group consisting of SEQ ID NOs:
- 3 to 5, or a sequence complementary thereto.
A method of preparing cells for administration to a patient in need of treatment of dystrophic epidermolysis bullosa, comprising:
- genetically modifying mesenchymal stem cells to produce type VII collagen, the cells having been obtained from the patient in need of treatment of dystrophic epidermolysis bullosa,
- wherein the genetically modified cells are capable of being administered to the patient.
A method of treating dystrophic epidermolysis bullosa, comprising:
- administering a therapeutically-effective amount of cells which produce type VII collagen to a patient in need thereof, the cells being mesenchymal stem cells having been previously obtained from the patient and genetically modified to produce type VII collagen.
Dependencies are inferred from the claim text.