Gene Therapy for Lumbar Disc Disease; An Overview of Animal and Human Studies

Document Type : Narrative Review


1 Department of Neurosurgery, Isfahan University of Medical Sciences, Isfahan, Iran

2 Isfahan Neurosciences Research Center, Alzahra Research Institute, Isfahan University of Medical Sciences, Isfahan, Iran

3 Functional Neurosurgery Research Center, Shohada Tajrish Neurosurgical Comprehensive Center of Excellence, Shahid Beheshti University of Medical Sciences,Tehran,Iran

4 Department of Tissue Engineering and Regenerative Medicine, School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, Tehran, Iran


Low back pain (LBP), a common disorder involving the muscles, nerves, and bones of the back, is associated with lumbar disc degeneration. It is a complex phenomenon, likely the result of a combination of biochemical and biomechanical factors that are known to occur in discs. However, the findings of previous studies have suggested that disc degeneration may be explained primarily by genetic influences. For this reason, scientists are interested in the use of genes/proteins for the treatment of disc degeneration. Protein-based therapies involve the administration of biologic factors into the intervertebral disc to enhance matrix synthesis, postpone degeneration, and prevent inflammation. These factors can be delivered by an intradiscal injection, alone or in combination with cells or tissue scaffolds, and by gene therapy. A systematic search for articles dated from 1990 to the present was performed to identify pertinent articles related to the topic of the role of gene therapy in treating intervertebral disc degeneration. Twenty-seven studies reported the use of at least one gene in treating this disease through gene therapy. Researchers have been performing phases I/II of a clinical trial on the treatment of disc degeneration with gene therapy since 2008. Recent studies have shown that gene therapy may have promise as a method of slowing down or preventing some of the changes seen in intervertebral discs. However, because the clinical trial is not complete and therefore the results are indeterminate, this method cannot be proffered as a replacement for surgery. It is hoped that definitive results of the possible effects of gene therapy on the human body will be acquired soon.