First Symposium on Natural Gene Therapy of the Skin

    March 2012 in “ Experimental dermatology ”
    Anna M. G. Pasmooij, Marcel F. Jonkman
    Image of study
    TLDR Natural gene therapy shows promise for treating skin disorders like epidermolysis bullosa.
    The first symposium on natural gene therapy of the skin, held in 2011, focused on revertant mosaicism (RM) in skin disorders like epidermolysis bullosa (EB), revealing RM in at least 35% of patients with specific EB mutations. The event discussed RM's occurrence, timing, mutation rates, and therapeutic potential, including the use of induced pluripotent stem cells (iPSCs) for diseases like recessive dystrophic epidermolysis bullosa (RDEB). Promising preliminary results in gene correction and skin grafting were presented, with interdisciplinary discussions and plans for a follow-up event in four years. The symposium was supported by the Dutch Butterfly Child Foundation and the European Society for Dermatological Research.
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