SBIR Phase I: Improved in Vivo Delivery of SiRNA

这个小型企业创新研究(SBIR)第一阶段研究项目旨在开发一种改进的方法，将小抑制性核糖核酸(SiRNA)输送到细胞中。所提出的方法将利用化学诱导的免疫偶联或将siRNAs与抗体直接连接作为改善siRNA进入细胞的机制。使用siRNA沉默感兴趣的基因已经成为调节基因表达的一个非常重要的机制，无论是在实验环境中还是在疾病中都是如此。目前在体内使用siRNA治疗的限制之一是细胞摄取率低。因此，提高靶细胞对siRNA摄取的方法将对科学界和医学界大有裨益。利用细胞摄取机制将这些有效的调节分子输送到细胞中，进一步打开了将特定的基因沉默分子用作体内治疗方式的可能性。

PARS SummaryThis Small Business Innovation Research (SBIR) Phase I research project aims to develop an improved method for the delivery of small inhibitory ribonucleic acids (siRNA) into cells. The proposed methodology will utilize chemically induced immuno-conjugates or direct linking of siRNAs to antibodies as the mechanism for improving siRNA delivery into the cells. Use of siRNA to silence genes of interest has become a very important mechanism to regulate gene expression both in experimental settings as well as in diseases. One of the current limitations to using siRNA therapy in vivo is the low uptake by the cells. Methods that improve siRNA uptake by target cells would therefore be of great benefit to the scientific and medical communities. The use of the cellular uptake mechanisms for the delivery of these potent regulatory molecules into cells further opens the possibility of using specific gene silencing molecules as therapeutic modalities in vivo.