Current Status of Biomedical Products for Gene and Cell Therapy of Recessive Dystrophic Epidermolysis Bullosa

被引:0
|
作者
Zorina, Alla [1 ,2 ]
Zorin, Vadim [1 ,2 ]
Isaev, Artur [1 ]
Kudlay, Dmitry [3 ,4 ]
Manturova, Natalia [5 ,6 ]
Ustugov, Andrei [5 ,6 ]
Kopnin, Pavel [7 ]
机构
[1] Artgen Biotech, Moscow 119333, Russia
[2] Skincell LLC, Moscow 119333, Russia
[3] IM Sechenov First Moscow State Med Univ, Sechenov Univ, Dept Pharmacol, Moscow 119991, Russia
[4] Lomonosov Moscow State Univ, Dept Pharmacognosy, Ind Pharm, Moscow 119992, Russia
[5] Pirogov Russian Natl Res Med Univ, Dept Plast & Reconstruct Surg Cosmetol & Cell Tech, Moscow 117997, Russia
[6] JSC Plast Surg & Cosmetol Inst, Moscow 125047, Russia
[7] NN Blokhin Natl Med Res Ctr Oncol, Sci Res Inst Carcinogenesis, Moscow 115522, Russia
基金
俄罗斯科学基金会;
关键词
epidermolysis bullosa; skin; treatment; gene and cell therapy; stem cells; gene delivery; study outcomes; MESENCHYMAL STEM-CELLS; BONE-MARROW-TRANSPLANTATION; RETROVIRAL DNA INTEGRATION; UMBILICAL-CORD BLOOD; VII COLLAGEN; STROMAL CELLS; MOUSE MODEL; FIBROBLAST THERAPY; CULTURED SKIN; COL7A1;
D O I
10.3390/ijms251910270
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
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.
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页数:29
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