换一批摘要近年来,随着分子遗传学研究的不断深入,越来越多的遗传性皮肤病致病基因得以明确,应用对致病基因的研究、载体构建、皮肤组织工程等的成果,将治疗基因转入先天性大疱性表皮松解症患者的皮肤细胞,成功在体外和(或)体内稳定表达具有功能的蛋白,使疾病相关的一些异常表现消失.目前先天性大疱性表皮松解症是皮肤病基因治疗研究最多的领域.
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abstractsRecently, with the continuing development in molecular genetics, pathogenic genes have been clarified for more and more inherited dermatoses. Furthermore, based on the knowledge about pathogenic genes, constructed vector and tissue engineering skin, some manifestations of congenital epidermolysis bullosa can be treated by transfecting therapeutic genes into skin cells of patients followed by stably expressing functional proteins in vivo or in vitro. Congenital epidermolysis bullosa is the hottest spot in gene therapy for dermatoses.
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