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Zorina, A.; Zorin, V.; Isaev, A.; Kudlay, D.; Manturova, N.; Ustugov, A.; Kopnin, P. Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa. Int. J. Mol. Sci. 2024, 25, 10270. https://doi.org/10.3390/ijms251910270

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Feature papers represent the most advanced research with significant potential for high impact in the field. A Feature Paper should be a substantial original Article that involves several techniques or approaches, provides an outlook for future research directions and describes possible research applications.

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Zorina A, Zorin V, Isaev A, Kudlay D, Manturova N, Ustugov A, Kopnin P. Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa. International Journal of Molecular Sciences. 2024; 25(19):10270. https://doi.org/10.3390/ijms251910270

Zorina A, Zorin V, Isaev A, Kudlay D, Manturova N, Ustugov A, Kopnin P. Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa. International Journal of Molecular Sciences. 2024; 25(19):10270. https://doi.org/10.3390/ijms251910270

Editor’s Choice articles are based on recommendations by the scientific editors of MDPI journals from around the world. Editors select a small number of articles recently published in the journal that they believe will be particularly interesting to readers, or important in the respective research area. The aim is to provide a snapshot of some of the most exciting work published in the various research areas of the journal.

Zorina, Alla, Vadim Zorin, Artur Isaev, Dmitry Kudlay, Natalia Manturova, Andrei Ustugov, and Pavel Kopnin. 2024. "Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa" International Journal of Molecular Sciences 25, no. 19: 10270. https://doi.org/10.3390/ijms251910270

Abstract: This detailed review describes innovative strategies and current products for gene and cell therapy at different stages of research and development to treat recessive dystrophic epidermolysis bullosa (RDEB) which is associated with the functional deficiency of collagen type VII alpha 1 (C7) caused by defects in the COL7A1 gene. The use of allogenic mesenchymal stem/stromal cells, which can be injected intradermally and intravenously, appears to be the most promising approach in the field of RDEB cell therapy. Injections of genetically modified autologous dermal fibroblasts are also worth mentioning under this framework. The most common methods of RDEB gene therapy are gene replacement using viral vectors and gene editing using programmable nucleases. Ex vivo epidermal transplants (ETs) based on autologous keratinocytes (Ks) have been developed using gene therapy methods; one such ET successively passed phase III clinical trials. Products based on the use of two-layer transplants have also been developed with both types of skin cells producing C7. Gene products have also been developed for local use. To date, significant progress has been achieved in the development of efficient biomedical products to treat RDEB, one of the most severe hereditary diseases. Keywords: epidermolysis bullosa; COL7A1; skin; treatment; gene and cell therapy; stem cells; gene delivery; study outcomes

Biomedical productslist

Zorina, A.; Zorin, V.; Isaev, A.; Kudlay, D.; Manturova, N.; Ustugov, A.; Kopnin, P. Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa. Int. J. Mol. Sci. 2024, 25, 10270. https://doi.org/10.3390/ijms251910270

Zorina, Alla, Vadim Zorin, Artur Isaev, Dmitry Kudlay, Natalia Manturova, Andrei Ustugov, and Pavel Kopnin. 2024. "Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa" International Journal of Molecular Sciences 25, no. 19: 10270. https://doi.org/10.3390/ijms251910270