Delivery nanotech gives RNAi potential a boost

Small interfering RNA (siRNA), a type of RNAi molecule, has the potential to be a powerful therapeutic agent and a step toward more personalized treatments. And while delivering the bits of genetic material to cells has proven challenging, a new nanotechnology might be on the right track.

Researchers at Washington University in St. Louis and Texas A&M University have developed nano-sized containers for siRNA to overcome the vast challenges that hinder drug targeting. The cationic shell-crosslinked knedel-like nanoparticles (cSCKs) react with both the drugs and cells in such a way that allow the transport of siRNA across the membrane. The current standard by which siRNA penetrates cell is through viral carriers, but these can have unintended consequences.

Like other RNAi drugs under development, once the siRNA can get into the cells, it uses a specific genetic code to "silence" or shut down parts of a patient's DNA that causes particular diseases. Companies like Alnylam Pharmaceuticals ($ALNY) are using similar delivery techniques as they approach a successful RNAi model.

"Shell-crosslinked nanoparticles are an attractive and versatile platform for the development of nucleic acid delivery agents because their synthetic design enables their size and shape, charge and buffering capacity, degradability, stealth character, and ligand presentation to be easily tailored for a particular target," Yuefei Shen and team wrote in the journal Nucleic Acid Therapeutics.

- here's the journal article