{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,16]],"date-time":"2026-08-16T07:05:53Z","timestamp":1786863953614,"version":"build-2736575974"},"reference-count":179,"publisher":"MDPI AG","issue":"19","license":[{"start":{"date-parts":[[2024,9,24]],"date-time":"2024-09-24T00:00:00Z","timestamp":1727136000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100006769","name":"Russian Science Foundation","doi-asserted-by":"publisher","award":["23-15-00433"],"award-info":[{"award-number":["23-15-00433"]}],"id":[{"id":"10.13039\/501100006769","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["IJMS"],"abstract":"<jats:p>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.<\/jats:p>","DOI":"10.3390\/ijms251910270","type":"journal-article","created":{"date-parts":[[2024,9,24]],"date-time":"2024-09-24T08:56:06Z","timestamp":1727168166000},"page":"10270","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa"],"prefix":"10.3390","volume":"25","author":[{"given":"Alla","family":"Zorina","sequence":"first","affiliation":[{"name":"Artgen Biotech, Moscow 119333, Russia"},{"name":"Skincell LLC, Moscow 119333, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vadim","family":"Zorin","sequence":"additional","affiliation":[{"name":"Artgen Biotech, Moscow 119333, Russia"},{"name":"Skincell LLC, Moscow 119333, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Artur","family":"Isaev","sequence":"additional","affiliation":[{"name":"Artgen Biotech, Moscow 119333, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dmitry","family":"Kudlay","sequence":"additional","affiliation":[{"name":"Department of Pharmacology, The I. M. Sechenov First Moscow State Medical University (The Sechenov University), Moscow 119991, Russia"},{"name":"Department of Pharmacognosy and Industrial Pharmacy, Lomonosov Moscow State University, Moscow 119992, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalia","family":"Manturova","sequence":"additional","affiliation":[{"name":"Department of Plastic and Reconstructive surgery, Cosmetology and Cell Technologies, Pirogov Russian National Research Medical University, Moscow 117997, Russia"},{"name":"JSC Plastic Surgery and Cosmetology Institute, Moscow 125047, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrei","family":"Ustugov","sequence":"additional","affiliation":[{"name":"Department of Plastic and Reconstructive surgery, Cosmetology and Cell Technologies, Pirogov Russian National Research Medical University, Moscow 117997, Russia"},{"name":"JSC Plastic Surgery and Cosmetology Institute, Moscow 125047, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2078-4274","authenticated-orcid":false,"given":"Pavel","family":"Kopnin","sequence":"additional","affiliation":[{"name":"Scientific Research Institute of Carcinogenesis, N. N. Blokhin National Medical Research Center of Oncology, Moscow 115522, Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,9,24]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1093\/bmb\/ldaa029","article-title":"Cellular therapy options for genetic skin disorders with a focus on recessive dystrophic epidermolysis bullosa","volume":"136","author":"Naso","year":"2020","journal-title":"Br. Med. 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