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Autologous keratinocytes transduced with a COL7A1-encoding retroviral vector is an ex vivo cell and gene therapy developed by Holostem for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). The therapy involves harvesting a patient's own keratinocytes and using a retroviral vector to insert a functional copy of the COL7A1 gene, which encodes type VII collagen. These genetically corrected cells are then grown into epidermal sheets and transplanted onto the patient's chronic wounds. By restoring the production of type VII collagen, the therapy aims to recreate the anchoring fibrils necessary for dermal-epidermal adhesion, thereby improving skin stability and promoting wound healing.
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