Research

Our research focuses on advancing macromolecule drug delivery technology by developing new materials. We are actively working in the following areas:

Gene therapeutics need to be successfully delivered to cell nuclei in order for gene expression to occur. One of the limitations of non-viral gene therapy is low delivery efficiencies due to poor intracellular trafficking. We are using rational materials design to create delivery vehicles that harness and exploit natural pathways to accomplish efficient intracellular delivery.

Nanoparticle-based drug delivery vehicles are typically ~50-200 nm in size. These vehicles can achieve extended circulation in the body and can be modified to target specific tissues. However, delivery is not optimal due to transport limitations. We are modeling these barriers and developing methods to increase interstitial transport of nanoparticles for applications in cancer therapy. We are also developing 3D culture platforms that can help to bridge the gap between 2D cell culture and animal studies.

Gene therapy is recognized as the most promising future treatment for hemophilia. One of the most popular approaches to hemophilia gene therapy is the restoration of clotting factor production to hepatocytes by introducing wildtype clotting factor genes to these cells. Although viral delivery systems have been primarily used, non-viral materials have many attractive qualities, including good safety profiles, low potential of immunogenicity, and ease in manufacturing and scale-up of materials. The objectives of this project are to systematically determine the physicochemical properties of nanoparticles that promote access to and specific uptake by hepatocytes in vivo and to develop formulation approaches for preparing nanoparticles with these properties for FIX gene delivery.

The delivery of exogenous nucleic acids to cells in the central nervous system is a powerful technique with applications in treatment of neurological disease. The purpose of this project is to develop nanoparticles that mediate efficient neuronal delivery. We are evaluating these novel nanoparticles as delivery vehicles in spinal injury models.

Localized gene delivery is an important technology for biomedical research. We are developing methods to spatially and temporally control gene delivery from biomaterials. In one application, we are incorporating gene delivery vehicles with tissue engineering scaffolds to deliver factors to promote functional tissue regeneration. In a second application, we are developing methods to pattern gene delivery vehicles on surfaces by non-covalent self-assembly.