Non-viral gene therapy.

Abstract

Non-viral gene therapies are currently under development that employ drug-delivery methods for targeting genes to selected cells in the body, where they express therapeutic gene products. Various methods have been described for non-viral gene therapy, ranging from the direct intramuscular injection of purified DNA to the systemic administration of formulations comprising DNA and lipids, proteins, peptides, or polymers. Products for non-viral gene therapies are designed both for direct administration to patients by conventional routes and for expression of a therapeutic product over a finite period of time in a manner similar to conventional medicines. Initial preclinical and clinical studies indicate that non-viral gene delivery methods exhibit safety profiles similar to conventional pharmaceutical or biological products. Clinical trials have been proposed, or are currently under way, to assess the applicability of non-viral gene therapy for a variety of disorders, including cystic fibrosis, cancer, and peripheral vascular disease. Non-viral techniques may soon allow gene therapy to be applied in clinical practice alongside conventional medicines for the treatment of common diseases.