Gene therapy applications for intervertebral disc degeneration

Abstract

Gene therapy is an emerging method of treating disease through the modification of a patient's genome toward a therapeutic goal. Gene transfer causes the recipient cells to synthesize new mRNA, which is encoded by the transferred gene, leading to the production of a specific target protein. The resultant protein may have several therapeutic biomodulatory effects. Several recent studies have suggested that gene therapy may have potential in the treatment of spinal disorders, especially intervertebral disc (IVD) degeneration. The successful transfer of therapeutic genes to target cells within the intervertebral disc by gene transfer in animal models reflects the rapid progress that is taking place in the field of gene therapy. The purpose of this article is to address several important technical topics, including 1) the basic theory of gene transfer; 2) characteristics of the target tissue (IVD cells); 3) gene transfer techniques; 4) current advances in IVD cell gene transfer; and 5) how these topics relate to the future clinical applications of gene therapy. Research in the field of gene therapy is growing at an exponential rate and has the potential to become a valuable treatment modality for the new millennium.