Research Project
6551561
DESCRIPTION (provided by applicant): Currently, treatment for chronic hepatitis C virus (HCV) infection is the systemic use of recombinant interferon-a (IFN-a). Unfortunately, IFN-a treatment typically requires frequent injections for an extended duration and is effective in only a minority of patients. Although the recent development of formulated versions of IFN-a have improved treatment, some patients still require weekly injections for over a year. Recent studies have demonstrated that long-term maintenance IFN-a therapy can slow progression of HCV-related liver inflammation and fibrosis, which often lead to chronic liver disease. Unfortunately, the cost, inconvenience, and side effects associated with bolus administration of IFN-a protein make it poorly suited for such indications. Gene-based production of IFN-a has been proposed as an alternative means of delivery. This approach could potentially provide stable, consistent serum IFN-a levels for several months, thereby minimizing the bolus kinetics associated with traditional IFN-a administration. Therefore, the goal of Phase I is to evaluate the basic feasibility of an IFN-a therapy based on delivery of plasmid DNA into muscle by electroporation. Successful development of a therapy capable of providing months of effective IFN-a production should provide a substantial improvement in the compliance, convenience, and cost of IFN-a therapy, potentially enabling treatment for a broader patient population.
