The experiments proposed in this Phase I application focus on developing a colloidal gold-based non-viral vector for targeted gene therapy by binding a cell-specific ligand and a DNA carrying protein to individual colloidal gold particles. The unique aspects of the proposed technology are: 1) the carrier system, colloidal gold, has a long history as a safe therapeutic itself, 2) the proposed vector system incorporates the newly described binding chemistries for carrying both a targeting ligand and a gene, and 3) the colloidal gold vector uses the biology of cell receptor internalization and the acidification of the endosome to efficiently deliver genetic therapies. This proposal is designed to achieve three goals. The short-term goal is to develop a new gene delivery kit This kit will be sold as a """"""""research-use- only"""""""" product to scientists to enable them to deliver their specific genes to their chosen cells. The broader use of this vector will aid CytImmune in achieving its intermediate goal: the building of the basic science foundation necessary to establish colloidal gold as a safe, effective gene delivery system for clinical use. Thus, CytImmune's long-term goal to establish this vector system as the first choice for clinical gene therapy may be realized.
The first commercial application of this research is the production of a new gene therapy kit for sale into the research-use-only market. The second commercial use is the development of a new, safe, cost-effective clinical gene delivery system.
