Novel nanoparticles for targeted delivery of siRNA in CNS diseases

M. Malhotra, C.-T. Duchesneau, S. Saha, S. Prakash
McGill University, CA

Keywords: nanoparticles, siRNA, targeted delivery, CNS diseases

Summary:

Therapeutic delivery across blood-brain barrier is challenging due to the neuroprotective mechanism of the brain. To address this issue, we have developed a synthetic polymeric nanoparticle formulation, that specifically delivers siRNA to the neuronal cells as a treatment for targeting CNS/neurodegenerative diseases. The developed nanoparticles possess features that aid them to cross the physiological barriers of the brain. The nanoparticle formulation features include, a size of <20 nm, a net positive surface charge, ability to be modified with any cell-targeting peptide, biodegradability, biocompatibility, with minimal/no toxicity to the neuronal cells as observed in experimental animal model.