Abstract:Recessive dystrophic epidermolysis bullosa (RDEB) is a blistering disorder caused by mutations in COL7A1 that encodes for the anchoring fibrils of type VII collagen. Our lab has demonstrated that non-viral gene delivery by polymer is able to restore the expression of type VII collagen (C7) to human RDEB skin and could overcome the possibility of generating an immune response when compared to viral vectors. Silencing caused by viral promoters is a major problem for an episomal non-viral gene therapy. To increas… Show more
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