Non-viral gene therapy vectors are particularly attractive for research and pharmaceutical development. The ideal vector would be of small size, be non-aggregating, targetable to specific cell types, highly efficient, and simple to formulate. The authors propose a novel approach in which DNA is condensed into small particles using a template polymerization approach. This approach allows flexibility in using both cationic and non-cationic compounds for condensation, and opens the possibility of incorporating targeting ligands during polymerization.
The specific aims of the proposal include: 1) Characterization of novel polymerization processes for reproducible preparation of small DNA-containing particles; 2) Physico-chemical characterization to determine which types of cationic compounds are optimal for production of stable, non-aggregating particles; and 3) Incorporation of specific ligands and endosome disrupting moieties in the particle formation process.
NOT AVAILABLE
