Review Focuses on Lipid Nanoparticles as Gene Therapy Carriers

A recent review article focused on the potential of solid lipid nanoparticles (SLNs) and nanostructured lipid carriers (NLCs) for gene therapy, including the main advances in their application for the treatment of ocular diseases, infectious diseases, lysosomal storage disorders, and cancer and current research for their future clinical application.

The review was written by investigators in the PharmaNanoGene: a pharmacokinetics, nanotechnology, and gene therapy research group at the University of the Basque Country. This team has been working on the design and evaluation of SLNs for treatment of diseases using gene therapy. They have been studying the relationship between formulation factors and the processes involving the intracellular internalization and disposition of the genetic material that condition the effectiveness of the vectors and which is essential in the optimization process.

The investigators wrote in the December 2016 issue of the European Journal of Pharmaceutics and Biopharmaceutics that non-viral vectors, including SLNs and NLCs, were less effective than viral vectors but much safer, and that their effectiveness has increased significantly in recent years. Lipid nanoparticles are able to overcome the main biological barriers for cell transfection, including degradation by nucleases, cell internalization intracellular trafficking, and selectively targeting to a specific cell type. They stressed that these nanoparticles were made up of biocompatible, biodegradable materials, that they were easy to produce on a large scale, that they could be sterilized and freeze-dried, and that they were very stable both in biological fluids and in storage.