Treatment for dystrophic epidermolysis bullosa

By expressing type VII chromatin collagen in genetically modified mesenchymal stem cells, the high manufacturing difficulty of existing treatments has been overcome, enabling effective treatment of dystrophic epidermolysis bullosa, reducing costs and improving patients' skin condition.

JP7833204B2Active Publication Date: 2026-03-19OSAKA UNIVERSITY
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Patents
Current Assignee / Owner
Filing Date
2024-07-31
Publication Date
2026-03-19

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Abstract

To provide a therapeutic agent for dystrophic epidermolysis bullosa.SOLUTION: The present invention provides a composition for use in the treatment of dystrophic epidermolysis bullosa, comprising a cell from a dystrophic epidermolysis bullosa patient that is genetically modified to produce type VII collagen through the introduction of a COL7A1 gene, wherein the cell is a mesenchymal stem cell and administered into a blister formed on the skin of the dystrophic epidermolysis bullosa patient.SELECTED DRAWING: None
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Citation Information

Patent Citations

  • Method for correcting a genetic sequence

    US20180142262A1

  • Gene therapy for recessive dystrophic epidermolysis bullosa using genetically corrected autologous keratinocytes

    WO2017120147A1

  • Compositions and methods for treatment of type vii collagen deficiencies

    WO2017161180A1

  • Materials and methods for treatment of dystrophic epidermolysis bullosa (DEB) and other collagen type vii alpha 1 chain (col7a1) gene related conditions or disorders

    WO2018154413A1

  • Use of coenzyme q10 formulations in the treatment and prevention of epidermolysis bullosa

    WO2018213502A1