Long-Term Survival of Type XVII Collagen Revertant Cells in an Animal Model of Revertant Cell Therapy

    Antoni Gostyński, Sara Llames, Marta García, M.J. Escámez, Lucía Martínez‐Santamaría, Miranda Nijenhuis, Álvaro Meana, Hendri H. Pas, Fernando Larcher, Anna M.G. Pasmooij, Marcel F. Jonkman, Marcela Del Río
    TLDR Revertant cell therapy shows promise for treating type XVII collagen deficiency, but better cell selection methods are needed.
    The study demonstrated the feasibility of revertant cell therapy for type XVII collagen deficiency in junctional epidermolysis bullosa. Revertant keratinocytes, which naturally corrected the genetic mutation, were harvested and used to create a bioengineered skin equivalent. Although there was a decrease in revertant cells during in vitro expansion, their percentage stabilized during skin equivalent production and remained stable after transplantation into an animal model. This indicated the long-term survival of revertant keratinocytes, suggesting that revertant epidermal stem cells were likely transplanted. The study highlighted the potential of revertant cell therapy but emphasized the need for methods to select C17 revertant cells to address in vitro depletion challenges.
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