Development of Genetic Therapies for the Hemidesmosomal Subtypes of Junctional Epidermolysis Bullosa

摘要

During the final period of the project, we were focusing on creating the model systems for the in vitro gene therapy experiments. The critical elements of this system are (1) the gene construct, (2) the gene delivery methodology, and (3) the in vitro skin model. We tested and compared the available delivery techniques by performing a series of transfection experiments using reporter gene systems (such as GFP) or antibiotic selection to assess efficiency. We worked extensively on development of the in vitro skin model, and succeeded in generating recombinant skin between keratinocytes and dermal cells in skin equivalents.