PJ5: Stem cell gene therapy

This is a therapeutic technology that enables the use of regeneration-inducing therapy for severe genetic diseases caused by gene deletion, etc. This is a product for radical regeneration-inducing cell therapy utilizing the proprietary stem cell culturing and preparation technology we have developed, with gene editing of the patient’s own stem cells performed outside the body to supply the genes that were deleted or mutated, after which the stem cells are returned to the patient’s body. Gene therapy generally targets stem cells of the affected organ, which means that gene therapy must be performed on different stem cells in the organ for each disease. Normal type VII collagen was genetically transferred into human mesenchymal stem cells and these cells were transplanted into the skin of epidermolysis bullosa model mice. The results demonstrated that human-derived type VII collagen functions normally in the skin of mice. In addition to pluripotency, mesenchymal stem cells have abilities such as immunomodulatory ability, and can have therapeutic effects in a variety of diseases, and thus the use of mesenchymal stem cells in gene therapy is expected to allow the treatment of a variety of genetic diseases.