Adenovirus-Mediated Gene Transfer to Treat Neurologic Disease

Within the last 10 years the development of tools used to manipulate DNA have shown promise in the generation of a new form of therapeutic intervention known as gene therapy. Gene therapy involves the introduction of genetic material into cells to replace or supplement defective genes or to induce the expression of a novel gene to help alleviate a disease. These manipulations can be done in cells either outside the body and transplanted back in (ex vivo gene therapy) or directly within a specific target organ (in vivo gene therapy). To target and transfer DNA or RNA to cells a variety of techniques have been developed, including the use of replication-defective viruses, lipid bilayers that encapsulate DNA (liposomes), and DNA-coated gold particles that are ballistically shot into cells (gene gun). Most of these topics will be discussed in articles to follow in this series.