GENE THERAPY FOR EPIDERMOLYSIS BULLOSA

该项目将开发皮肤基因 治疗交界性大疱性表皮病（JEB），同时研究 皮肤生物学的关键相关特征。 这个的目标 项目重点是进一步表征基底膜区 与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 交付方法。 我们将努力开发有效的 将治疗性基因长期递送至皮肤的方法 作为JEB基因治疗的基础， 在治疗人类遗传性皮肤病中的应用。

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.