siRNA Delivery Using a Smart Triblock Copolymer Carrier

描述（由申请人提供）：本提案的目的是开发一种基于聚（乙二醇）、聚（丙硫醚）和siRNA（PEG-PPS-S-siRNA）的自组装三嵌段共聚物的小干扰RNA（siRNA）递送系统。 此外，将合成聚合物PEG-PPS-PEG-RGD并用于通过整合素介导的内吞作用靶向内化。 在体内或体外将siRNA有效递送至哺乳动物细胞具有使基因组中的任何基因沉默的潜力，这对于基因功能的研究、遗传性疾病的治疗和组织形成的增强具有意义。 该提议的核心假设是三嵌段共聚物PEG-PPS-S-siRNA可以被定制以利用细胞器和细胞途径中的化学差异来实现siRNA的有效递送。 该系统将被开发为体内和体外腹部粘连预防研究的核心技术，并将使目前无法进行的许多研究成为可能。

DESCRIPTION (provided by applicant): The goal of this proposal is to develop a delivery system for small interfering RNA (siRNA) based on the self-assembling triblock copolymers of poly (ethylene glycol), poly (propylene sulfide) and siRNA (PEG-PPS-S-siRNA). Additionally, the polymer PEG-PPS-PEG-RGD will be synthesized and used to target internalization via integrin-mediated endocytosis. Efficiently delivering siRNA to mammalian cells in vivo or in vitro has the potential to silence any gene in the genome, which has implications for the study of gene function, the treatment of inherited disorders and the augmentation of tissue formation. The central hypothesis underlying this proposal is that the triblock copolymer PEG-PPS-S-siRNA can be tailored to take advantage of chemical differences in the cell organelles and of cellular pathways to achieve efficient delivery of siRNA. This system will be developed to serve as a central technology for studies in abdominal adhesion prevention in vivo and in vitro, and will enable numerous studies that are not currently possible.