Ex vivo gene therapy cures a blistering skin disease |
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Authors: | Featherstone Carol Uitto Jouni |
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Institution: | 1. Department of Orthopaedics, The Second Hospital of Shanxi Medical University, Shanxi Key Lab of Bone and Soft Tissue Injury Repair, Taiyuan 030001, China;2. Department of Orthopaedics, Warren Alpert Medical School of Brown University/Rhode Island Hospital, Providence, RI 02903, USA;3. Foot and Ankle Orthopaedic Surgery Center, Beijing Tongren Hospital, Capital Medical University, Beijing 100730, China |
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Abstract: | A recent publication that describes gene therapy treatment of a patient with an inherited blistering skin disease, epidermolysis bullosa, demonstrates for the first time that gene therapy can cure a disease of solid tissue. The treatment relies on ex vivo transduction of autologous epidermal stem cells with a normal copy of the defective gene, followed by reconstitution of the patient's skin with epithelial sheets that are grown from these genetically corrected cells. This approach holds promise for treatment not only of inherited disorders of the skin but also of other solid tissues that are becoming amenable to tissue engineering. |
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