TARGETING GENES TO CELLS RELEVANT TO CYSTIC FIBROSIS

描述（改编自应用程序）：长期目标 该项目旨在开发囊性纤维化的基因治疗方法 （CF）。 该项目有两个具体目标。 第一个目标是确定 表达囊性纤维化跨膜传导调节因子的细胞 （CFTR）在肺中。 这将通过使用单克隆抗体来完成， CFTR。 第二个具体目标是开发分子工具， 需要在这些细胞中传递和稳定表达基因。 组织 多聚免疫球蛋白受体（pIgR）的特异性表达将是 用于靶向非降解内化的大分子， 基底外侧面 抗pIgR抗体的Fab段共价偶联 连接到DNA的聚阳离子将用于引导外源DNA进入 想要的细胞 将确定靶向的特异性， 优化的DNA的摄取和表达。

DESCRIPTION (adapted from the application): The long term objective of this project is to develop methods for gene therapy of cystic fibrosis (CF). The project has two specific aims. The first aim is to identify the cells that express the cystic fibrosis transmembrane conductance regulator (CFTR) in the lung. This will be done by using monoclonal antibodies to CFTR. The second specific aim is to develop the molecular tools that are needed to deliver and stably express genes in those cells. The tissue specific expression of the polymeric immunoglobulin receptor (pIgR) will be used to target non-degradative internalization of macromolecules from the basolateral surface. A Fab fragment of antibody to pIgR covalently coupled to a polycation link to DNA will be used to direct foreign DNA into the desired cells. The specificity of the targeting will be determined and uptake and expression of the DNA optimized.