Research ArticleSKIN DISEASE

Genetically corrected iPSCs as cell therapy for recessive dystrophic epidermolysis bullosa

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Science Translational Medicine  26 Nov 2014:
Vol. 6, Issue 264, pp. 264ra165
DOI: 10.1126/scitranslmed.3010083

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iPSC-Derived Cell Therapy for Epidermolysis Bullosa

Recessive dystrophic epidermolysis bullosa (RDEB) is a genetic condition where even the soft touch of a cotton sheet can cause severe skin blistering and pain. Patients with RDEB often spend their lives in bandages, with no treatment options at present. Now, Wenzel et al. offer a cell therapy that would use induced pluripotent stem cells from patients to treat the wounded areas. The authors took fibroblasts from the tails of mice with or without RDEB (with or without mutated Col7a1) and used these to create iPSC lines. The iPSCs were genetically corrected to express type VII collagen and then differentiated into keratinocytes (skin cells) or fibroblasts. When iPSC-derived fibroblasts were delivered back to the animals with RDEB, only the genetically corrected cells created skin layers that expressed type VII collagen and were resistant to mechanical friction (no blistering). Genetically repaired iPSCs, therefore, offer a viable cell therapy for RDEB if the methods of gene correction can be made safe for use in humans.

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