Gene Therapy: Nucleic Acid Delivery Using Lipid Nanoparticles

Lipid nanoparticle (LNP) is the state-of-the-art approach for nucleic acid delivery and gene therapy. However, only <2% of the nucleic acid delivered by the standard LNP formulation is released in the cytosol of cells for gene regulation. We are synthesizing novel lipids and polymers to replace the lipid components in the LNP formulation to increase gene delivery efficiency. This technology can be applied to vaccine development and gene therapy for genetic disorders.