SBIR Phase I: Improved in Vivo Delivery of SiRNA

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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.