RNA interference (RNAi) is an important biological mechanism to silence certain genes, thereby suppressing the related protein expression. From a therapeutic perspective, small interfering RNA (siRNA) has an enourmous therapeutic potential as it could be used to downregulate expression of proteins that cause disease symptoms. Moreover, it has been reported that such siRNA therapeutics could remain active for several months rather than hours or days as for small molecules and antibodies. However, siRNA is quickly degraded in vivo and also needs to enter into the cell to become active. Therefore, formulation or chemical modification of siRNA is required to transform siRNA into a therapeutic formulation.
Within this project we will further develop our recently discovered cationic polymer delivery platform for siRNA by strengthening the patent application, developing an optimized cationic polymer siRNA delivery platform and by generating in vivo proof-of-concept for gene silencing.