The potential of gene therapy for the treatment of disc degeneration.

Abstract

Research in biologic methods of treating disc degeneration is still in its infancy. Many different strategies are being evaluated, but the gene therapy strategy stands out because of its potential for long-term efficacy. Choosing the correct gene for use in gene therapy is critically important. Of the many different classes of potentially therapeutic genes, the regulatory genes hold the most promise. Of the different gene therapy delivery methods, the most work has been performed with viral vectors, either ex vivo or in vivo. Current research now is turning toward in vivo experiments in rabbits. Efficacy and safety will be demonstrated first with smaller animal models. Beyond that, nonhuman primate experiments demonstrating efficacy and safety will be the penultimate step before initiation of human studies.