Non-viral siRNA delivery vectors: dendritic molecular transporter and molecular transporter nanovectors for target gene silencing‡

Viable non-viral delivery vectors have been utilized for the intracellular delivery of siRNA duplexes to result in complete target gene silencing. Biologically active siRNA duplexes were attached to a dendritic molecular transporter (MT) or a molecular transporter-polymeric nanoparticle conjugate (MT-NP) via a thiol exchange reaction utilizing a dithiol pyridyl linker. The MT and the MT-NP delivery systems resulted in significant reduction of the target protein (lamin A/C) investigated for a range of conjugate concentrations. Both non-viral systems have proven effective for the delivery of siRNA into the cytosol of live cells and specifically the development of the MT-NP delivery system provides opportunities for targeted delivery of siRNA into selected cell types.