GENE THERAPY FOR EPIDERMOLYSIS BULLOSA

拟议的计划项目将开发皮肤基因 交界性大疱性表皮松解症的边研究边治疗 皮肤生物学的关键相关特征。这样做的目的是 项目重点是进一步确定基底膜区域的特征 与水疱性人类皮肤病有关的蛋白质，以及 皮肤基因调控策略、干细胞生物学和基因 投递方式。我们将努力开发出有效的 长期向皮肤输送治疗基因的方法 作为JEB基因治疗的基础和系统的 在人类遗传性皮肤病治疗中的应用。

The proposed program project will develop cutaneous gene therapy for junctional epidermolysis bullosa (JEB) while studying key relevant features of cutaneous biology. The goals of this project focus on further characterizing basement membrane zone proteins in relationship to blistering human skin disease, as well as on cutaneous gene regulation strategies, stem cell biology and gene delivery methods. We shall attempt to develop effective approaches for long term delivery of therapeutic genes to the skin as a foundation for gene therapy for JEB and for systematic application in the treatment of human genetic skin disease.