Gene Therapy Applications for Spine Fusion

Objective. This article reviews the potential utilization of various growth factors to enhance spinal fusion and outlines the principles of gene therapy and its application to spinal fusion surgery. Summary of Background Data. Gene therapy offers an exciting new way to potentially deliver growth factors locally in a targeted fashion with physiologic doses. In its current definition, gene therapy is defined as the use of nucleic acid transfer, either RNA or DNA, to treat or prevent a disease. The scope of gene therapy has expanded beyond its initial application as a method of replacing genetic defects, and its potential to facilitate spinal fusions is currently being evaluated. Conclusions. Gene therapy strategies for spine fusion are appealing because the setting is uniquely suited for genetic manipulation. The intervention is locally applied. Only a short duration of transgene response by the cells is necessary to establish a spine fusion, and a variety of osteoinductive growth factors have been identified and are available for use. Attempts at spine fusion using gene therapy in the lower animals have been successful using both in vivo and ex vivo approaches. Before human clinical trials can be established, further testing is required in more challenging animal models of bone induction such as nonhuman primates. Should a successful clinical program of gene therapy for spine fusion be established, the use of autograft and its associated morbidities could be eliminated. In fact, gene therapy offers the potential for minimally invasive applications that could bypass the need for an open procedure altogether. It is likely that gene therapy will be a powerful therapeutic tool for the spine surgeon in the new millennium.