Design and development of targeted gene delivery systems

The knowledge of the genome and proteome will result in enormous opportunities in the identification of new therapeutic molecules which will ultimately have a major impact on human health. The new types of pharmaceuticals will be DNA and RNA pieces, small peptides, large proteins and recombinant or subunit vaccines. The science and technology of the delivery and targeting of the products of the genome and proteome is the next crucial link in the development of new approaches for the treatment and prevention of diseases. The objective in gene therapy is the introduction of a missing or defective gene into the cell nucleus where the encoded protein can be expressed. During the transfection process the DNA crosses several barriers such as the cellular, lysosomal, and nuclear membranes prior to expression.This research program will develop efficient non-viral gene delivery systems for application through the skin and cellular barriers to achieve both local and systemic expression of therapeutic proteins for dermatological diseases (localized scleroderma, atopic dermatitis,wound healing problems)as well as for delivery into the blood circulation for conditions such as anemia that may develop as a result of cancer treatment or a disease condition such as HIV.